Novartis' programme providing free access to its spinal muscular atrophy (SMA) gene therapy Zolgensma is being scaled back to a dozen countries worldwide, according to the company.
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
Roche and PTC Therapeutics' Evrysdi for spinal muscular atrophy (SMA) has been approved by the FDA for younger children with the rare disease, extending its use to include infants less than
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.
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