Site Search

Results

Search Results : 1201

1 2 … 80 81 82 … 120 121

Clear All Filters

Fedratinib for Myelofibrosis

Myelofibrosis is a cancer of the bone marrow in which the marrow is replaced by scar tissue. The early stages of myelofibrosis may be asymptomatic in some people while others may have severe symptoms from the onset. As the bone marrow becomes more scarred, it is less able to produce blood cells leading to enlargement […]

Mitapivat for treating pyruvate kinase deficiency

Mitapivat is currently in clinical development for the treatment of adult patients with pyruvate kinase deficiency (PKD) who have regular blood transfusions and those who do not have regular blood transfusions. PKD is a genetic blood disorder caused by low levels of the enzyme pyruvate kinase (PK). Low levels of PK result in a deficiency […]

Mobocertinib for locally advanced or metastatic NSCLC

Mobocertinib (TAK-788) as a monotherapy is in clinical developmentfor the treatment oflocallyadvanced or metastatic non-small cell lung cancer (NSCLC). NSCLC makes up the majority of lung cancers in the UK. In locally advanced or metastaticNSCLC(StageIIIB orIV),the cancer has spread beyond the lung which was initially affected. Symptoms of lung cancer include a persistent cough, shortness […]

Mosunetuzumab for relapsed or refractory B-cell follicular lymphoma – third-line and greater

Mosunetuzumab is in clinical development for the treatment of patients with relapsed or refractory B-cell follicular lymphoma (FL). FL is a type of slow growing blood cancer that affects white blood cells. In FL, the affected white blood cells start to multiply in an abnormal way and begin to aggregate in certain parts of the […]

Vestronidase alfa (UX-003) for mucopolysaccharidosis type VII (MPS 7; Sly syndrome)

Mucopolysaccharidosis type VII (MPS 7) is an extremely rare, inherited condition caused by a deficiency of an enzyme called beta-glucuronidase. Normally the enzyme breaks down mucopolysaccharides, however as it is lacking the breakdown does not take place. The incomplete broken down mucopolysaccharides remain stored in cells in the body causing progressive damage. Death in the […]

Ixazomib Citrate for Newly Diagnosed Multiple Myeloma – maintenance therapy following autologous stem cell transplant

Multiple myeloma (MM) is a rare, incurable cancer of the plasma cells in the bone marrow which is the spongy tissue found at the centre of some bones. MM is the second most common haematological cancer in the UK. Symptoms of MM vary but may include bone pain, fractures, body weakness, malaise, bleeding, anaemia and […]

bb2121 for Relapsed and Refractory Multiple Myeloma

Multiple myeloma (MM) is a rare, incurable cancer of the plasma cells in the bone marrow. Bone marrow is the spongy tissue found at the centre of some bones, which produces blood cells for the body. Plasma cells are normally produced in a controlled way but in cases of MM, large amounts of abnormal plasma […]

MTX-110 for newly-diagnosed diffuse intrinsic pontine glioma

MTX-110 is in clinical development for newly-diagnosed diffuse intrinsic pontine glioma (DIPG). DIPG is a high grade childhood brain tumour. The cause is unknown. DIPG develops in a part of the brain stem known as the pons, which controls essential body functions including heartbeat, breathing, eyesight and balance. The symptoms of DIPG may include; abnormal […]

Mirikizumab for the treatment of moderately to severely active ulcerative colitis

Mirikizumab is in clinical development for the treatment of adult patients with moderately to severely active ulcerative colitis (UC). UC is a long-term condition where the colon and rectum (parts of the bowel), become inflamed. Small ulcers can develop on the colon’s lining, which can cause rectal bleeding and recurring diarrhoea. The symptoms of ulcerative […]

Migalastat for Fabry disease in children aged 12 to 15 years

Migalastat is in clinical development for the treatment of Fabry disease for children aged 12 to 15 years old. Fabry disease is a rare genetic disorder caused by a defective gene (the GLA gene) in the body. In most cases, the defect in the gene causes a deficient quantity of the enzyme alpha-galactosidase A. This […]