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public registry

Our clinical study is listed on ClinicalTrials.gov, a website maintained by the US National Library of Medicine (NLM) at the US National Institutes of Health (NIH). ClinicalTrials.gov describes each study in specialised medical terms and lists the medical centers where study participants would receive the new treatment.

general anaesthesia

The procedure to inject the investigational therapy into the spinal cord will be made under general anaesthesia. A person under general anaesthesia will not move, feel pain, or remember anything about the procedure. Doctors use a special anaesthetic medicine to induce this deep sleep state. The study team monitors the patient closely until the procedure is complete and the patient wakes up naturally a few minutes later.

AS symptoms

The symptoms of AS are wide ranging and vary for each individual. This is reflected in the wide range of measurements that will be used in this study. These measurements will help us understand the impact of the investigational therapy on symptoms relating to seizures, sleep disruption, anxiety and aggression.

Angelman Syndrome

Angelman Syndrome (AS) is a rare genetic disease diagnosed in 1 per 12,000 to 20,000 births. Disease-causing genetic abnormalities prevent the brain from producing a protein called UBE3A, which is critical for the brain to develop normally.

Investigational therapy

An investigational therapy is a therapy that is being reviewed by health authorities under carefully controlled conditions. A team of healthcare professionals ensure that patients receive the therapy at specific times, and at fixed intervals. The team is especially vigilant for signs of an unexpected side effect of the therapy. Side effects, expected or otherwise, are reported to health authorities.

Acute pain

An unpleasant feeling of pain that comes on quickly and lasts for a short time. It can happen following surgery or trauma, or another health condition, and acts often as a warning to the body to seek help.

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Adenocarcinoma

A cancer that starts in cells of the body that produce fluids and lubricants, such as the gut.

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Adjuvant treatment

A treatment that is often given after surgery to kill any remaining cancer cells and lower the risk that the cancer will come back. Adjuvant treatment may include chemotherapy, radiotherapy, hormone treatment, targeted therapy, or biological therapy.

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Advanced cancer

Cancer that may have begun to spread to surrounding tissues or lymph nodes and is normally larger in size than tumours in patients without advanced cancer. In some cases, advanced cancer also describes a tumour that has come back after being treated.

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Age-related macular degeneration

A medical condition that causes blurred or reduced vision in the centre of one or both eyes caused by damage to an area of the retina in the back of the eye called the macula.

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AMD

A medical condition that causes blurred or reduced vision in the centre of one or both eyes caused by damage to an area of the retina in the back of the eye called the macula.

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Amniocentesis

A test done during pregnancy to take a small amount of fluid from around the baby in the womb to test for conditions in the growing baby.

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Anaemia

A condition where a person has a reduced amount of haemoglobin or number of red blood cells, which can lower the ability to carry enough oxygen around the body.

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Angiogenesis

The growth of blood vessels. Tumour angiogenesis is the growth of new blood vessels that cancers need in order to grow.

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Anti-inflammatory

A medicine or other method used to reduce inflammation, e.g. in the form of redness, swelling, or pain (inflammation).

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Antibody

Antibodies are created by cells of the immune system and are carried around the body by blood and lymph. They stick, for example, to cells or parts of cells in an individual’s body to determine if the cells are good or bad. Antibodies that stick to bacteria and viruses, which cause infections, help the body to destroy the bacteria and viruses, protecting the body against infection. Certain types of antibodies (called monoclonal antibodies), made in the laboratory instead of the body, can detect bad cells in the body such as cancer cells and help to destroy them.

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Antibodies

Antibodies are created by cells of the immune system and are carried around the body by blood and lymph. They stick, for example, to cells or parts of cells in an individual’s body to determine if the cells are good or bad. Antibodies that stick to bacteria and viruses, which cause infections, help the body to destroy the bacteria and viruses, protecting the body against infection. Certain types of antibodies (called monoclonal antibodies), made in the laboratory instead of the body, can detect bad cells in the body such as cancer cells and help to destroy them.

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Antiviral medicine

A type of medicine designed to treat infections caused by viruses.

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Asthma

A common long-term lung condition caused by swelling (inflammation) of the airways that causes difficulty with breathing.

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Autoimmune disease

A term used to describe a group of diseases where the immune system of an organism attacks it's own healthy cells or structures in the body by mistake. Autoimmune diseases can affect different parts of the body such as the gut (for example Crohn’s disease and ulcerative colitis), the joints (for example rheumatoid arthritis) or the skin (for example psoriasis).

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Best supportive care

Care that focuses on relieving the symptoms caused by serious illnesses (for example cancer)

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Biological therapy/treatment

A therapy based on products from a living organism and used in the prevention, diagnosis, or treatment of cancer and other diseases. Biological drugs include for instance antibodies, interleukins, and vaccinations.

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Biopsy

A common surgical procedure that involves taking a small sample of cells or tissue so it can be examined under a microscope. The most common types of biopsies are incisional biopsies (where only a sample of tissue is removed), excisional biopsies (where an entire lump or suspicious area is removed) and needle biopsies (where a sample of tissue or fluid is removed with a needle).

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Blinding

Blinding means that people involved in clinical trials do not know whether they are given the new drug being tested, an older drug that is known to work, or a placebo that does not contain any active drug. This makes it easier to understand if a new drug works well.

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Bone marrow transplant

A procedure where a patient receives healthy blood-forming cells (called stem cells) to replace their own stem cells that have been destroyed by disease, or by radiation or high doses of anticancer drugs that are given as part of their treatment. A bone marrow transplant may be autologous (by using a patient’s own stem cells that were collected from the marrow and saved before treatment), allogeneic (by using stem cells donated by someone who is not an identical twin), or syngeneic (by using stem cells donated by an identical twin).

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Bypassing agent

These are medicines that are used for haemophilia. They work around (or bypass) inhibitors that stop another medicine from working to help the body form a normal blood clot.

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Cancer

When people are healthy, cells grow and multiply at a steady controlled rate, which is necessary for bodies to work well (for example to heal after illness or injury). Sometimes a change, or alteration, to the DNA that gives instructions on how cells should behave can randomly happen when cells divide. These alterations (also called ‘mutations’) can cause cells to grow and multiply out of control, eventually forming a group of abnormal cancer cells known as a tumour.

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CIT

A type of therapy that encourages or suppresses the body’s own immune system to fight cancer cells.

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Cancer of unknown primary site

The term used when cancer is found in a patient’s body, but the original (or primary) place the cancer started is not known. At the time of diagnosis, CUP presents as metastasis at different places across the body.

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CUP

The term used when cancer is found in a patient’s body, but the original (or primary) place the cancer started is not known. At the time of diagnosis, CUP presents as metastasis at different places across the body.

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Capsule endoscopy

An examination where a patient swallows a small capsule that contains a tiny camera and light. This takes photos of the inside of a patient’s gut as it passes through for their doctor to examine.

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Carrier

An individual person who is capable of passing on a genetic alteration associated with a disease, but who may or may not display disease symptoms themselves.

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Checkpoint inhibitors

To detect cells in the body that are bad or defective, the immune system uses specific molecules (or checkpoints) on some immune cells to start the attack on bad cells. Cancer cells sometimes find ways to use these checkpoints to avoid being attacked by the immune system. Checkpoint inhibitors are medicines that stop the cancer cells protecting themselves.

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Chemotherapy

Drugs that stop cancer growing, either by killing the cancer cells or by stopping them from dividing.

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Chorionic villus sampling

A test done during pregnancy to take a small number of cells from the placenta connecting the mother to her baby in the womb to test for conditions in the growing baby.

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Chromosome

Genes are packaged as chromosomes (a long chain of genes folded up tightly to fit in a cell). There are 23 pairs of chromosomes in each cell of an individual without any genetic disorders. One-half of the pair comes from the mother, the other half from the father.

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Chronic

A term used to describe something that gets worse slowly and continues for a long period of time. It can describe a disease, or patients’ symptoms and signs. The opposite of chronic is acute.

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Chronic kidney dysfunction

A term that describes life-long disorders of the kidney.

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Chronic metabolic diseases

A term that describes the combination of increased blood pressure, high blood sugar, excess body fat around the waist, and abnormal cholesterol or triglyceride levels, increasing the risk of heart disease, stroke and diabetes. These conditions are life-long and cannot be cured. However, with effective management (for example treatments or lifestyle changes), people can continue to live a full life.

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Chronic obstructive pulmonary disease

A term that describes a group of lung diseases including emphysema (the destruction of lung tissue that allows people to breathe), chronic bronchitis (inflammation of the bronchial tubes that carry air to and from the lungs) and refractory (non-reversible) asthma. These conditions are life-long.

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COPD

A term that describes a group of lung diseases including emphysema (the destruction of lung tissue that allows people to breathe), chronic bronchitis (inflammation of the bronchial tubes that carry air to and from the lungs) and refractory (non-reversible) asthma. These conditions are life-long.

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Chronic pain

Ongoing pain lasting more than 12 weeks.

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Chronic respiratory disease

A term that describes all life-long conditions affecting the organs and tissues that make breathing and transport of oxygen and other gases around the body possible.

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Cirrhosis

Liver scarring that results from previous damage, such as from an infection or from drinking too much alcohol. This scarring can cause problems with the way the liver works and this can lead to further problems.

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Clinical trial

Clinical trials are medical research studies that involve people. The aim of a clinical trial is to test new drugs to see how well they work in helping people feel better, or in helping a disease to improve or stop getting worse. Clinical trials also test whether new drugs are safe and if they have any side effects. If clinical trials show that the new drug works well and does not have too many side effects, the company can apply to official organisations in different countries to ask if the drug can be given to other people with the disease who are not in clinical trials

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Cognitive dysfunction

The impairment of the intellectual functions such as thinking, remembering and reasoning that can be subclinical or bad enough to get in the way of daily activities.

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Colorectal cancer

The colon and rectum make up the large intestine, which is the final part of the gut (or the digestive or gastrointestinal) system. Colorectal cancer (also known as bowel cancer) is a broad term meaning cancer that develops in the colon or rectum.

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CRC

The colon and rectum make up the large intestine, which is the final part of the gut (or the digestive or gastrointestinal) system. Colorectal cancer (also known as bowel cancer) is a broad term meaning cancer that develops in the colon or rectum.

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Colonoscopy

A technique where a special tube with a camera is inserted through a patient’s anus to view the rectum and colon to check for any unusual growths or inflammation in the gut. A sample (or biopsy) of any unusual growths may also be taken to check whether there is any sign of colorectal cancer.

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CT

Also known as computerised tomography. A type of scan that uses X-rays and a computer to create detailed images of the inside of the body.

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Corticosteroids

A steroid hormone that can be made by the body or in a laboratory. Corticosteroids have many different effects in the body, and are used to treat many different conditions. They may be used as hormone replacement, to suppress the immune system, and to treat some side effects of cancer and its treatment.

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Crohn’s disease

An autoimmune bowel disease that can cause inflammation (swelling, redness, and pain) anywhere in the gut, but most often in part of the small intestines called the ‘ileum’ or in the large intestine, also known as the colon.

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Cystic fibrosis

A condition caused by a damaged gene that affects the movement of salt and water in and out of cells. This, combined with repeated infections, can cause a build-up of thick, sticky mucus in the body's airways. This particularly affects the lungs and digestive system, causing a wide range of challenging complications all over the body.

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DNA or deoxyribonucleic acid

Also known as deoxyribonucleic acid. Genetic instructions used in the development, growth, functioning and reproduction of all known living organisms and many viruses.

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Diabetes mellitus

A term used to describe a group of disorders that cause a person’s blood sugar to become too high. This disease occurs when the body does not make enough insulin or does not use insulin the way it should.

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Diarrhoea

Frequent, loose and watery bowel movements.

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Diffuse large B-cell lymphoma

The most common type of lymphoma and the most common type of aggressive non-Hodgkin lymphoma, which spreads all over lymph nodes rapidly.

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DLBCL

The most common type of lymphoma and the most common type of aggressive non-Hodgkin lymphoma, which spreads all over lymph nodes rapidly.

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Double blinding

Double blinding means that both the researchers running the clinical trial and the people taking part in the clinical trial do not know which drug a person is given until after the clinical trial has finished.

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Dry age-related macular degeneration

Dry age-related macular degeneration is the most common form of age-related macular degeneration, occurring in around nine out of ten patients. It is caused by the layers of the macula, in the back of the eye, slowly becoming thinner as old cells die without being replaced by the body. This causes the macula to gradually stop working properly with vision in the centre of the eye slowly being lost.

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Endoscopy

Insertion of a thin tube with a camera, either through the mouth (to view the oesophagus and stomach) or through the anus (to view the rectum and colon).

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EGFR

Also known as epidermal growth factor receptor. The protein found on the surface of some cells. When epidermal growth factor binds to EGFR the cells divide, creating new cells. An alteration in the EGFR gene can lead to cancer.

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Progesterone receptor

The estrogen and progesterone receptors are proteins found on the cells of the female reproductive tissue, some other types of tissue, and some cancer cells. The sex hormones estrogen and progesterone will bind to the receptors and can cause the cells to grow. When cancers have estrogen receptors they are called ‘ER-positive’ or ‘ER+’. When cancers have progesterone receptors they are called ‘PR-positive’ or ‘PR+’. Checking the amount of estrogen or progesterone receptors on some types of cancer cells may help to plan treatment.

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Estrogen receptor

The estrogen and progesterone receptors are proteins found on the cells of the female reproductive tissue, some other types of tissue, and some cancer cells. The sex hormones estrogen and progesterone will bind to the receptors and can cause the cells to grow. When cancers have estrogen receptors they are called ‘ER-positive’ or ‘ER+’. When cancers have progesterone receptors they are called ‘PR-positive’ or ‘PR+’. Checking the amount of estrogen or progesterone receptors on some types of cancer cells may help to plan treatment.

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ER-positive

The estrogen and progesterone receptors are proteins found on the cells of the female reproductive tissue, some other types of tissue, and some cancer cells. The sex hormones estrogen and progesterone will bind to the receptors and can cause the cells to grow. When cancers have estrogen receptors they are called ‘ER-positive’ or ‘ER+’. When cancers have progesterone receptors they are called ‘PR-positive’ or ‘PR+’. Checking the amount of estrogen or progesterone receptors on some types of cancer cells may help to plan treatment.

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ER+

The estrogen and progesterone receptors are proteins found on the cells of the female reproductive tissue, some other types of tissue, and some cancer cells. The sex hormones estrogen and progesterone will bind to the receptors and can cause the cells to grow. When cancers have estrogen receptors they are called ‘ER-positive’ or ‘ER+’. When cancers have progesterone receptors they are called ‘PR-positive’ or ‘PR+’. Checking the amount of estrogen or progesterone receptors on some types of cancer cells may help to plan treatment.

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PR-positive

The estrogen and progesterone receptors are proteins found on the cells of the female reproductive tissue, some other types of tissue, and some cancer cells. The sex hormones estrogen and progesterone will bind to the receptors and can cause the cells to grow. When cancers have estrogen receptors they are called ‘ER-positive’ or ‘ER+’. When cancers have progesterone receptors they are called ‘PR-positive’ or ‘PR+’. Checking the amount of estrogen or progesterone receptors on some types of cancer cells may help to plan treatment.

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PR+

The estrogen and progesterone receptors are proteins found on the cells of the female reproductive tissue, some other types of tissue, and some cancer cells. The sex hormones estrogen and progesterone will bind to the receptors and can cause the cells to grow. When cancers have estrogen receptors they are called ‘ER-positive’ or ‘ER+’. When cancers have progesterone receptors they are called ‘PR-positive’ or ‘PR+’. Checking the amount of estrogen or progesterone receptors on some types of cancer cells may help to plan treatment.

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European Medicines Agency

European Union agency whose role is to protect and promote public health by overseeing the use of medicinal products in Europe Union member states. The EMA’s scientific committees review the evidence from clinical trials and make a recommendation on whether a medicine should be made available for the treatment of a particular disease. The decision is based upon several factors, including how well the medicine works and how safe the medicine is.

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EMA

European Union agency whose role is to protect and promote public health by overseeing the use of medicinal products in Europe Union member states. The EMA’s scientific committees review the evidence from clinical trials and make a recommendation on whether a medicine should be made available for the treatment of a particular disease. The decision is based upon several factors, including how well the medicine works and how safe the medicine is.

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Extranodal disease

When cancerous lymphocytes (white blood cells) travel to sites of the body other than the lymph nodes (for example the lungs, liver, blood, bone marrow, kidneys, brain, and spinal cord).

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Factor 8/9

Also known as factor VIII/IX. Haemophilia is caused by an alteration (a defect or change) in a person’s genes. People with haemophilia A have a defect in a gene called factor 8 and people with haemophilia B have a defect in a gene called factor 9. In both cases, the affected gene is either missing or does not work.

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Factor 8

Also known as factor VIII. Haemophilia is caused by an alteration (a defect or change) in a person’s genes. People with haemophilia A have a defect in a gene called factor 8. The affected gene is either missing or does not work.

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Factor 9

Also known as factor IX. Haemophilia is caused by an alteration (a defect or change) in a person’s genes. People with haemophilia B have a defect in a gene called factor 9. The affected gene is either missing or does not work.

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Food and Drug Administration

US agency whose role is to protect and promote public health in the US. The FDA decides if evidence from clinical trials has shown that a new medicine works well enough and is safe enough to be approved for use in the treatment of a particular disease.

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FDA

US agency whose role is to protect and promote public health in the US. The FDA decides if evidence from clinical trials has shown that a new medicine works well enough and is safe enough to be approved for use in the treatment of a particular disease.

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Fibrin

A protein that forms a sticky mesh across injured blood vessels to create a clot and stop bleeding, similar to a plaster placed over a wound.

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Fistulas

Abnormal channels or passageways that form from one internal organ to another, or to the outside surface of the body. They can occur in any part of the body, but they are most common in the digestive system.

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Follicular lymphoma

The most common type of low-grade (slow-growing) non-Hodgkin lymphoma and the second most common type of lymphoma. Follicular lymphoma usually begins in the lymph nodes, but can spread throughout the body.

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Gene

Genes are the instructions for how to build all known living organisms from bacteria to humans. They are pieces of DNA inherited from parents and contain all the information needed to make people who they are – from the colour of someone’s eyes to their blood type.

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Genetic carrier

A person who is capable of passing on a genetic alteration associated with a disease, but who may or may not display disease symptoms themselves.

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Genetics

The way that characteristics of living organisms (from the colour of someone’s eyes to their blood type) are passed from a parent to their child.

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Genomic profile

This gives information about specific genes, including how they are different from one person to another in a certain type of disease. A genomic profile may be used to help diagnose a disease or to learn how the disease may get worse without treatments or get better with different treatments.

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Glucorticoids

A type of corticosteroid medication that is used to treat a range of diseases caused by inflammation, including rheumatoid arthritis, by suppressing the body’s immune system.

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Haematuria

Blood in the urine.

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Hepatocellular carcinoma

The most common type of liver cancer. HCC begins when alterations (defects or changes) occur in hepatocyte (specialised liver cell) DNA. These alterations affect the DNA instructions and can lead to uncontrolled cell growth and eventually the formation of a tumour.

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HCC

The most common type of liver cancer. HCC begins when alterations (defects or changes) occur in hepatocyte (specialised liver cell) DNA. These alterations affect the DNA instructions and can lead to uncontrolled cell growth and eventually the formation of a tumour.

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Hepatitis

A term that describes inflammation of the liver. It is usually the result of liver damage caused by a viral infection or by drinking too much alcohol.

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Hepatitis virus

There are three types of hepatitis virus called A, B and C. Long-term infection with hepatitis B or C can cause liver cirrhosis (a scarring of the tissues that can cause problems with the way the liver works).

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HER2

Also known as human epidermal growth factor receptor 2. HER2 is a protein involved in normal cell growth. It can be made in larger than normal amounts by some types of cancer cells, including breast cancer, and cause cancer cells to grow more quickly. When cancers have more HER2 than normal, they are called ‘HER2-positive’ or ‘HER2+’. Checking the amount of HER2 on some types of cancer cells may help to plan treatment.

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HER2+

Also known as human epidermal growth factor receptor 2. HER2 is a protein involved in normal cell growth. It can be made in larger than normal amounts by some types of cancer cells, including breast cancer, and cause cancer cells to grow more quickly. When cancers have more HER2 than normal, they are called ‘HER2-positive’ or ‘HER2+’. Checking the amount of HER2 on some types of cancer cells may help to plan treatment.

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HER2-positive

Also known as human epidermal growth factor receptor 2. HER2 is a protein involved in normal cell growth. It can be made in larger than normal amounts by some types of cancer cells, including breast cancer, and cause cancer cells to grow more quickly. When cancers have more HER2 than normal, they are called ‘HER2-positive’ or ‘HER2+’. Checking the amount of HER2 on some types of cancer cells may help to plan treatment.

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Hormone treatment

A treatment that uses medicines to block or lower the amount of hormones in the body to slow down or stop the growth of cancer.

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Host cell

A cell that is infected by a virus or another type of microorganism

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HIV

Also known as human immunodeficiency virus. A virus that damages the cells in a person’s immune system and weakens their ability to fight everyday infections and disease.

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Immune cells

White blood cells that protect the body from foreign (or harmful) substances, cells and viruses by creating an immune response.

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Immune tolerance induction

A method where medicine is given regularly over a period of time until the body is trained to recognise the treatment product without reacting (without forming an immune response) to it.

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Investigator-initiated studies

Investigator Initiated Studies (IIS) are clinical trials that are designed and run by researchers who don't work for a pharmaceutical company. In these trials, a pharmaceutical company such as Roche may be a supporting collaborator.

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ITI

A method where medicine is given regularly over a period of time until the body is trained to recognise the treatment product without reacting (without forming an immune response) to it.

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Immune system

The process that protects the body from foreign (or harmful) substances, cells and viruses by creating white blood cells to fight the bad cells detected.

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Immunosuppression

A partial or complete suppression of the immune system that can leave a person at a high risk of infection, which they may be less able to fight off.

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Immunotherapy

Immunotherapy uses substances either made naturally by the body or manmade in a laboratory to boost the immune system in order to stop or slow cancer cell growth.

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Implant

A term used to describe a device that is surgically inserted into the body to deliver a medicine, replace a bit of the body that is damaged or missing, or to monitor/measure what is happening in the body.

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Inflammatory bowel disease

A condition that causes inflammation of the digestive system. Inflammation is the body’s reaction to an injury, infection or irritation and can cause redness, swelling and pain. There are two common types of IBD, which are called Crohn’s disease and ulcerative colitis. While these can have the same symptoms, they can affect different parts of the digestive system.

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IBD

A condition that causes inflammation of the digestive system. Inflammation is the body’s reaction to an injury, infection or irritation and can cause redness, swelling and pain. There are two common types of IBD, which are called Crohn’s disease and ulcerative colitis. While these can have the same symptoms, they can affect different parts of the digestive system.

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Influenza

Influenza is a relatively common infection of the airways that causes fever, intense aching and cold-like symptoms, a general feeling of weakness and pain in the muscles, and often headaches. It is caused by the influenza virus.

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Flu

The flu is a relatively common infection of the airways that causes fever, intense aching and cold-like symptoms, a general feeling of weakness and pain in the muscles, and often headaches. It is caused by the influenza virus.

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Influenza virus

The highly infectious virus responsible for causing influenza. There are three types of influenza virus known as type A, B and C.

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Intracranial haemorrhage

A bleed inside the skull.

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Intravenous infusion

An intravenous infusion is where medicine is given directly into the bloodstream over time in a controlled, steady manner. This is usually done in a clinic as it involves a plastic tube inserted into a vein so that the amount and speed of the medicine can be carefully controlled by a healthcare professional over a period of time.

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Large cell carcinoma

Cancer cells that are large and round with big nuclei (the part of the cell that contains all of its genetic information).

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Lymphatic system

The tissues and organs that produce, store, and carry white blood cells that fight infections and other diseases. This system includes the bone marrow, spleen, thymus, lymph nodes and lymphatic vessels (a network of thin tubes that carry lymph and white blood cells).

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Lymphocyte

Lymphocytes help protect the body from infection. They travel continuously around the body through a network known as the lymphatic system, which includes the bone marrow, spleen, thymus, lymph nodes, and lymphatic vessels (a network of thin tubes that carry lymph and white blood cells).

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Lymphoma

A cancer that starts in the vessels of the lymphatic system.

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Macula

The macula is an area in the back of the eye, near the centre of the retina, which is the part of the eye that has the best vision.

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Metastatic cancer

Metastatic cancer is a tumour that has spread from where it originally began to another organ or tissue (for example breast cancer cells that move to the bone).

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Monoclonal antibody treatment

Antibodies are created by cells of the immune system and are carried around the body by blood. They stick to cells or parts of cells in an individual’s body to work out if the cells are good or bad. Antibodies that stick to bad bacteria and viruses, which cause infections, help the body to destroy the bacteria and viruses, protecting the body against the infection. Certain types of antibodies (called monoclonal antibodies), made in the laboratory instead of the body, can detect other bad cells in the body such as cancer cells and help to destroy them.

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MRI

Also known as magnetic resonance imaging. A type of scan that uses strong magnetic fields and radio waves linked to a computer to produce detailed images of the inside of the body.

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mTOR inhibitor

A medicine that blocks mTOR, which can cause cancer cells to die.

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Multiple sclerosis

A disease that affects a patient’s central nervous system (the brain and spinal cord), which can eventually lead to disability.

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Mutation, more universally referred to as an alteration

A sudden or inherited change in the DNA pattern that makes up a gene, so that the gene is different from what is found in healthy cells of humans or animals, or any other living organism. An alteration can induce cancer.

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Myelin

A substance that surrounds nerve fibres, insulating them like electrical wires, so that electrical signals pass through them easily.

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Neoadjuvant therapy/treatment

Treatment given before surgery to shrink a tumour.

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Neovascular age-related macular degeneration

A form of age-related macular degeneration that can cause a patient to lose their eyesight very quickly, sometimes within a few weeks. This is caused by unhealthy blood vessels growing under the macula (part of the retina at the back of the eye) where they would not normally grow. The new unhealthy vessels can cause swelling and bleeding under the macula causing scarring and loss of vision.

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Wet age-related macular degeneration

A form of age-related macular degeneration that can cause a patient to lose their eyesight very quickly, sometimes within a few weeks. This is caused by unhealthy blood vessels growing under the macula (part of the retina at the back of the eye) where they would not normally grow. The new unhealthy vessels can cause swelling and bleeding under the macula causing scarring and loss of vision.

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Nephrectomy

Removal of the kidney by surgery.

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Neuromuscular disorders

A term used to describe a group of disorders that affect the how the muscles work.

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Non-small-cell lung cancer

The most common type of lung cancer. There are three main types of NSCLC (adenocarcinoma, squamous cell carcinoma and large cell carcinoma), which are determined from the way the tumour cells appear when looked at under a microscope.

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NSCLC

The most common type of lung cancer. There are three main types of NSCLC (adenocarcinoma, squamous cell carcinoma and large cell carcinoma), which are determined from the way the tumour cells appear when looked at under a microscope.

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Non-Hodgkin lymphoma

A type of cancer that starts in white blood cells called lymphocytes, which help protect the body from infection. Lymphocytes become abnormal and are unable to fight infections. The abnormal lymphocytes tend to collect in the lymph nodes and spleen, causing them to swell and form cancerous tumours.

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NHL

A type of cancer that starts in white blood cells called lymphocytes, which help protect the body from infection. Lymphocytes become abnormal and are unable to fight infections. The abnormal lymphocytes tend to collect in the lymph nodes and spleen, causing them to swell and form cancerous tumours.

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Non-steroidal antiinflammatory

An anti-inflammatory medicine used to reduce swelling (inflammation).

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Optical coherence tomography

A scan that uses light and a computer to provide a cross sectional image of the retina.

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Osteoporosis

A condition that is most common in older women, where bones become increasingly weak and fragile, causing them to eventually crumble and break.

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Pancolitis

A severe form of ulcerative colitis that has spread throughout the entire large bowel/intestine.

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Peripheral vision

The ability to see objects and movement outside of the direct line of vision (side vision).

Placebo

A placebo is used in clinical trials to help researchers understand if the new drug works well. It does not contain any active drug but looks the same, and is given the same way, as the new test drug.

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Platinum-doublet chemotherapy

A combination of two cancer-killing drugs that contain platinum, which are given together and used to treat certain forms of lung cancer.

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Pneumonia

An infection of the lungs that is caused by a variety of microorganisms, including bacteria, viruses and fungi, which produces swelling of the air sacs in one or both lungs. The air sacs may fill with fluid or pus, causing a cough, fever, chills and difficulty breathing.

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Progesterone receptor

The progesterone and estrogen receptors are proteins found on the cells of the female reproductive tissue, some other types of tissue, and some cancer cells. The sex hormones progesterone and estrogen will bind to the receptors and can cause the cells to grow. When cancers have progesterone receptors they are called ‘PR-positive’ or ‘PR+’. When cancers have estrogen receptors they are called ‘ER-positive’ or ‘ER+’. Checking the amount of progesterone or estrogen receptors on some types of cancer cells may help to plan treatment.

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PR-positive

The progesterone and estrogen receptors are proteins found on the cells of the female reproductive tissue, some other types of tissue, and some cancer cells. The sex hormones progesterone and estrogen will bind to the receptors and can cause the cells to grow. When cancers have progesterone receptors they are called ‘PR-positive’ or ‘PR+’. When cancers have estrogen receptors they are called ‘ER-positive’ or ‘ER+’. Checking the amount of progesterone or estrogen receptors on some types of cancer cells may help to plan treatment.

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PR+

The progesterone and estrogen receptors are proteins found on the cells of the female reproductive tissue, some other types of tissue, and some cancer cells. The sex hormones progesterone and estrogen will bind to the receptors and can cause the cells to grow. When cancers have progesterone receptors they are called ‘PR-positive’ or ‘PR+’. When cancers have estrogen receptors they are called ‘ER-positive’ or ‘ER+’. Checking the amount of progesterone or estrogen receptors on some types of cancer cells may help to plan treatment.

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Estrogen receptor

The progesterone and estrogen receptors are proteins found on the cells of the female reproductive tissue, some other types of tissue, and some cancer cells. The sex hormones progesterone and estrogen will bind to the receptors and can cause the cells to grow. When cancers have progesterone receptors they are called ‘PR-positive’ or ‘PR+’. When cancers have estrogen receptors they are called ‘ER-positive’ or ‘ER+’. Checking the amount of progesterone or estrogen receptors on some types of cancer cells may help to plan treatment.

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ER+

The progesterone and estrogen receptors are proteins found on the cells of the female reproductive tissue, some other types of tissue, and some cancer cells. The sex hormones progesterone and estrogen will bind to the receptors and can cause the cells to grow. When cancers have progesterone receptors they are called ‘PR-positive’ or ‘PR+’. When cancers have estrogen receptors they are called ‘ER-positive’ or ‘ER+’. Checking the amount of progesterone or estrogen receptors on some types of cancer cells may help to plan treatment.

More info

ER-positive

The progesterone and estrogen receptors are proteins found on the cells of the female reproductive tissue, some other types of tissue, and some cancer cells. The sex hormones progesterone and estrogen will bind to the receptors and can cause the cells to grow. When cancers have progesterone receptors they are called ‘PR-positive’ or ‘PR+’. When cancers have estrogen receptors they are called ‘ER-positive’ or ‘ER+’. Checking the amount of progesterone or estrogen receptors on some types of cancer cells may help to plan treatment.

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Proctitis

Inflammation of the lining of the rectum.

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Prophylactic treatment

A treatment to protect a person from a disease (or the symptoms) they have, or may be exposed to. For example, prophylactic treatment can be given to patients with haemophilia (who are at risk of severe bleeds) to prevent their bleeds before they happen.

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Protein

A long chain of very small molecules called amino acids that are organised into both simple and complex structures, and form almost everything in a living organism, from hair and skin to enzymes and antibodies.

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Radiotherapy

A treatment where the body is exposed to radiation to damage cancer cells and ultimately kill them.

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Randomised

A method in clinical trials using chance (like flipping a coin) to split a group of patients into two or more smaller groups. Patients in the different groups are given different drugs so one can be compared with another.

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Recombinant DNA

A form of artificial DNA that is created when pieces of DNA from different sources are joined together.

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Remission

When the signs and symptoms of cancer or other diseases disappear. A remission can be temporary or permanent.

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Renal cell carcinoma

The most common type of kidney cancer. About nine out of ten of kidney cancers are RCC.

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RCC

The most common type of kidney cancer. About nine out of ten of kidney cancers are RCC.

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Resection

Partial or complete removal of an organ, tissue or structure by surgery.

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Rheumatoid arthritis

An inflammatory disease of the joints of the body, which causes painful swelling.

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Sarcoma

A cancer that starts in the muscle or bones.

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Seizure disorder

A condition where a person has multiple attacks causing convulsions, muscle spasms and sometimes loss of consciousness.

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Small-cell lung cancer

A type of lung cancer. The type of lung cancer is determined from the way the tumour cells appear when examined under a microscope.

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Sickle cell anaemia

A condition where a person’s red blood cells do not last as long as normal red blood cells. The cells get stuck in blood vessels due to an abnormal ‘sickle-like’ shape, resulting in a reduction in the number of blood cells. This is a serious and life-long condition where red blood cells cannot carry enough oxygen around the body.

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Side effects

An unwanted effect of a drug or medical treatment. For example, some chemotherapies can cause hair loss.

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Sponsor

The organization or person who initiates the study and who has authority and control over the study.

Spontaneous bleeding

Bleeding that occurs in someone who has not had any damage or trauma to their body because they have a disorder that stops their blood clotting when it should.

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Squamous cell carcinoma

A cancer that starts in the flat cells that cover the airway surface of the lungs

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Stem cell transplant

A procedure where a patient receives healthy blood-forming cells (called stem cells) to replace their own stem cells that have been destroyed by disease, or by the radiation or high doses of anticancer drugs that are given as part of their treatment. A bone marrow transplant may be autologous (by using a patient’s own stem cells that were collected from the marrow and saved before treatment), allogeneic (by using stem cells donated by someone who is not an identical twin), or syngeneic (by using stem cells donated by an identical twin).

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Strictures

Areas of the bowel that have narrowed due to the growth of scar tissue from repeated inflammation (swelling) and healing.

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Stricturoplasty

The removal of strictures in the digestive system by surgery.

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Targeted therapy

A type of treatment that uses drugs to find and attack specific types of cancer cells with less harm to normal cells. Some targeted therapies block the action of certain molecules involved in the growth and spread of cancer cells. Other types of targeted therapies help the immune system to kill cancer cells or deliver toxic substances directly to cancer cells to kill them. Targeted therapy may have fewer side effects than other types of cancer treatment.

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TNM staging

A tumour grading system. These refer to the size of the tumour (T), whether the cancer has spread to nearby lymph nodes (N) and whether the cancer has spread (or metastasised) to other parts of the body (M).

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Triple-negative breast cancer

A type of cancer where tumour cells do not have receptors for the hormones estrogen and progesterone or the protein HER2. This type of cancer cannot be treated with standard hormone treatment or targeted treatment. Patients would usually be given surgery or chemotherapy.

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TNBC

A type of cancer where tumour cells do not have receptors for the hormones estrogen and progesterone or the protein HER2. This type of cancer cannot be treated with standard hormone treatment or targeted treatment. Patients would usually be given surgery or chemotherapy.

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Trivalent vaccine

A vaccine that contains three types of dead, inactive virus strains that cannot cause infection.

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Tyrosine kinase

Tyrosine kinases are a part of many cell functions, including cell growth and dividing. They may be too active or high levels may be found in some types of cancer cells, so blocking them may help to keep cancer cells from growing.

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Tyrosine kinase inhibitors

These drugs block tyrosine kinases, which are part of the specific communication process that controls the basic activities of cells that can cause cancer to grow uncontrollably.

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Ulcerative colitis

A condition that causes inflammation in the inner lining of the rectum and the lower colon. As well as inflammation, patients with ulcerative colitis can also develop ulcers on the lining of their colon that can bleed and produce mucus, resulting in diarrhoea (frequent loose stools or bowel movements) and bleeding from the rectum.

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Vaccination

An injection including an inactive virus, or a small part of an active virus, that cannot cause infection by itself in a normal person. The injection helps the body identify the virus as ‘bad’ so if the body is attacked by the real virus in the future it can protect itself against the virus and disease.

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VEGF

Also known as vascular endothelial growth factor inhibitors. VEGF is a protein that is involved in creating new blood vessels, and treatment with VEGF inhibitors stops new unwanted blood vessels from growing.

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Virus

A simple microorganism that infects cells and may cause disease.

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Trial Length

The trial length is calculated as the whole duration of the trial, from the (estimated / actual) start date to the (estimated / actual) end date from CT.gov. The period of time the patient is participating in the trial may be shorter.

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mTOR

Also known as mammalian/mechanistic target of rapamycin. A protein that helps control several cell functions, including cell division and survival, and may be more active in some types of cancer cells than it is in normal cells.

Leukemia

A type of cancer that affects the blood and bone marrow.

Solid tumours

A tumour that does not contain any liquid or cysts.

Acute myeloid leukemia

A particular type of leukemia that progresses quickly and affects the myeloid cells. The myeloid cells are white blood cells that fight bacterial infections, defend the body against parasites and prevent the spread of tissue damage.

AML

A particular type of leukemia that progresses quickly and affects the myeloid cells. The myeloid cells are white blood cells that fight bacterial infections, defend the body against parasites and prevent the spread of tissue damage.

Acute lymphocytic leukemia

A particular type of leukemia that progresses quickly and affects the lymphocytes. The lymphocytes are white blood cells that fight viruses.

Neuroblastoma

A type of cancer that develops from specialised nerve cells and mainly affects babies and young children.

Lumbar puncture

A procedure where a needle is inserted into the spine to collect spinal fluid and/or inject drugs into the spinal fluid.

Investigator-Initiated Trial

Investigator Initiated Trials are clinical trials that are designed and run by researchers who don’t work for a pharmaceutical company. These trials may be partly funded by Roche.

Motor neurons

What are motor neurons? Motor neurons are nerve cells that control muscle movement. Upper motor neurons send messages from the brain to the spinal cord, and lower motor neurons send messages from the spinal cord to the muscles. They form part of the neuromuscular system and are essential for day-to-day activities like breathing, holding your head up, walking and even holding a book.