spinal muscular atrophy

News

Scholar Rock has won its first product approval, on its second attempt, as the FDA gives a green light to its spinal muscular atrophy drug Isembyld.

News

All newborns in England will be screened for rare muscle-wasting disease spinal muscular atrophy from 2027, in a landmark move for patients.

News

Novartis has secured EU approval for Itvisma, a new formulation of its spinal muscular atrophy gene therapy usable in a broader patient population.

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.

News

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