European regulators have recommended approval of Itvisma, a gene therapy for spinal muscular atrophy, marking another step forward in the development of advanced treatments for rare neuromuscular diseases. The Committee for Medicinal Products for Human Use adopted a positive opinion on 23 April 2026, supporting the granting of a marketing authorisation for the therapy. As…
Category: Spinal muscular atrophy
FDA approves higher dose nusinersen for spinal muscular atrophy
The US Food and Drug Administration has approved a higher dose regimen of nusinersen for the treatment of spinal muscular atrophy, marking a significant development nearly a decade after the therapy was first introduced. The updated dosing approach increases the concentration of nusinersen administered during both the initial loading phase and ongoing maintenance treatment. It…
Scotland pilots newborn screening for spinal muscular atrophy
Scotland has become the first nation in the UK to introduce routine newborn screening for spinal muscular atrophy, marking a significant step forward in early diagnosis and treatment of the rare genetic condition. The screening will be delivered through the existing heel prick blood spot test offered to all newborns, as part of a two…
Evrysdi Tablet for Spinal Muscular Atrophy (SMA)
Genentech’s Evrysdi tablet is first and only tablet for SMA approved by the FDA Evrysdi is the only non-invasive disease-modifying SMA treatment and is approved in over 100 countries – Evrysdi tablet can be stored at room temperature and offers the same demonstrated efficacy and safety as the currently available oral solution – New tablet…
