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.
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
NICE has rejected routine NHS funding for Roche's Evrysdi (risdiplam) for spinal muscular atrophy drug in draft guidance, a decision which doesn't come as a surprise to patient association
Sanofi is still plugging away with its oral BTK inhibitor tolebrutinib in multiple sclerosis, but has decided to abandon efforts to develop the drug for neuromuscular diso
In the newest episode of the pharmaphorum podcast, editor-in-chief Jonah Comstock speaks with Jameka Hill, senior director of clinical trial health equity at Moderna.