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Emapalumab is a type of drug called a monoclonal antibody, under development for the treatment of primary HLH in children. It acts by neutralising IFNγ activity produced by over‐stimulated cells of the immune system. It is administered as an intravenous infusion and may be used in addition to current treatment.

Voxelotor is as an oral, once-daily therapy that is being developed for treatment of SCD. It is designed to work by helping haemoglobin hold onto more oxygen as the red blood cells travel through the body.

Myelodysplastic syndromes (MDS) are a group of blood disorders, in which the bone marrow does not produce enough healthy red blood cells, white blood cells and/or platelets. Faulty cells often die earlier than normal cells and the body destroys some abnormal cells, leaving the patient with low blood counts. This can result in anaemia (not …

Eltrombopag (Revolade) is a new drug to treat severe aplastic anaemia. Eltrombopag is taken by mouth and makes the body produce more platelet cells by activating a hormone called thrombopoietin.

Hereditary angioedema (HAE) is a rare inherited condition that causes swelling of the tissues (angioedema). These swellings happen suddenly and can affect any part of the body, including the hands and feet, arms and legs, trunk, intestines, genital organs, face, tongue, neck and airway. Cinryze is a drug that is already used to treat adolescents …

Myelofibrosis is a rare condition affecting the bone marrow and most people with this condition do not have any symptoms. Bone marrow is where blood cells are made. In myelofibrosis, scar tissue builds up inside the bone marrow and blood cells are not made properly. Occasionally the spleen (which functions as a blood filter) may …

Caplacizumab is a drug that is being given for the treatment of thrombotic thrombocytopenic purpura. The drug is injected under the skin and given alongside the current treatment of plasma exchange.

Begelomab is a new drug for people whose first treatment for GvHD has failed. Begelomab is given in a drip (directly into a vein). Begelomab is currently being studied to see how well it works and whether they it is safe to use in people with GvHD. If begelomab is licensed for use in the …