Concern about the safety of gene therapies has been thrust into the spotlight again with the news that two patients treated with Novartis' spinal muscular atrophy (SMA) treatment Zolgensma
Spinal muscular atrophy (SMA) is a devastating, genetic neuromuscular disease caused by a lack of a functional SMN1 gene that results in the progressive and irreversible loss of motor neuro
Spinal muscular atrophy (SMA) therapy Spinraza has been a key earner for Biogen in recent years, but with sales now heading into reverse, the company is looking to extend its franchise.
Roche's oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with
Roche has highlighted new data with its oral treatment for spinal muscular atrophy (SMA) – Evrysdi – showing its benefits when given to pre-symptomatic babies with the rare disease.
The FDA has relaxed a clinical hold on clinical trials of Novartis' intrathecal formulation of spinal muscular atrophy (SMA) gene therapy Zolgensma, allowing a new phase 3 trial to get unde
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