spinal muscular atrophy

News

A new FDA approval means Novartis now has a gene therapy option for almost everyone with spinal muscular atrophy (SMA), regardless of their age.

News

Biogen's efforts to defend its SMA business have been dealt a setback after the FDA declined to approve a high-dose version of Spinraza.

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Unresolved issues at a manufacturing plant operated by Novo Nordisk have led to a complete response letter for Scholar Rock's SMA drug apitegromab.

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Biogen buys Alcyone, bringing a partnered technology for delivery of antisense drugs into the central nervous system in-house.

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Biogen is looking to the future of its spinal muscular atrophy (SMA) business, taking a candidate offering once-yearly dosing into phase 3.

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Should treatment of neurodegenerative disorder SMA with Roche's Evrysdi start even before birth? One case study suggests so.