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New gene therapy for the treatment of children with ultra-rare immune disorder recommended for appro

Orphan-designated Strimvelis to offer treatment option for patients with ADA-SCID who have no suitable stem cell donor

The European Medicines Agency (EMA) has recommended granting a marketing authorisation in the European Union (EU) for a new gene therapy for the treatment of patients with adenosine-deaminase-deficient severe combined immunodeficiency (ADA-SCID), who have no matching donor for a stem cell transplant. Children born with this disorder have virtually no immunity to fight off everyday bacterial, fungal or viral infections.

ADA-SCID is an ultra-rare immune disorder, caused by a faulty gene inherited from both parents that stops the production of adenosine deaminase. Without this enzyme, the body is unable to break down a toxic substance called deoxyadenosine. The toxin builds up and destroys infection-fighting lymphocytes.

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