Dr Haitham Elbashir, Consultant Pediatric Neurorehabilitation at Al Jalila Children’s Hospital, said, "Zolgensma is a transformative therapy that offers children with SMA the chance to regain strength ...

Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy appeared at risk. Sarepta has reported three patient deaths related to its gene therapies ...

For example, Novartis' gene therapy Zolgensma for spinal muscular atrophy has also caused liver toxicity and death, but the benefit of that treatment is clear.

Zolgensma, which is used to treat spinal muscular atrophy, can cause side effects. Learn about its more common, mild, and serious side effects and how to manage them.

Biogen's early-stage spinal muscular atrophy drug salanersen showed safety, motor improvements and 70% reduction in neurodegeneration markers after one-year dosing.

Biotech Biogen’s Spinraza follow-up helps children with SMA who didn’t benefit from Zolgensma By James Waldron Jun 25, 2025 8:04am ...