neurological disease

Policy paper available on unmet needs in Neuromyelitis Optica Spectrum Disorders (NMOSD) in Europe

NewsPar Ngangta Mbaidoum 24 mars 2022

The Global Alliance for Patient Access has made available a policy paper on unmet needs in Neuromyelitis Optica Spectrum Disorders (NMOSD) in Europe, published in multiple languages. A rare autoimmune condition of the central nervous system, NMOSD causes deterioration to a person’s optic nerve and spinal cord. It affects over 10,000 people in Europe but…

EMA recommends approval for first gene therapy to treat children with rare inherited neurological disease

NewsPar Ngangta Mbaidoum 28 mai 2021

Based on evidence from a single-arm clinical trial, the European Medicines Agency (EMA) has recommended granting a marketing authorisation in the European Union for the gene therapy SKYSONA™ for the treatment of children with cerebral adrenoleukodystrophy (CALD), a severe form of a rare inherited neurological disease. SKYSONA™, developed by bluebird bio, is a one-time treatment which can…

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neurological disease

Policy paper available on unmet needs in Neuromyelitis Optica Spectrum Disorders (NMOSD) in Europe

EMA recommends approval for first gene therapy to treat children with rare inherited neurological disease

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