US FDA approves Bluebird bio’s gene therapy for rare neurological disorder

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Sept. 16 – The U.S. Food and Drug Administration has approved bluebird bio’s gene therapy for the treatment of a rare neurological disorder, the company announced Friday evening.

« SKYSONA is the first FDA-approved therapy that slows the progression of neurological dysfunction in boys » with early and active cerebral adrenoleukodystrophy (CALD), the company said in a statement, claiming that CALD is a « devastating neurodegenerative disease and deadly”.

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(Reporting by Mrinalika Roy and Jaiveer Shekhawatin in Bengaluru; Editing by Sriraj Kalluvila, Devika Syamnath and William Mallard)


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