Spinal Muscular Atrophy: A Rare but Treatable Disease of the Nervous System
Treatments & Research
Treatments & Research
Although there’s currently no cure for SMA, drug treatments are now available.
These pages tell you about any NHS-funded drug treatments, any in the pipeline, and other related topics. Whatever the treatment, it remains vital that anyone who has SMA also has appropriate care and management based on assessment by and discussion with their clinical team. Though some recommendations are now changing, the internationally agreed Standards of Care for SMA remains a useful reference point.
Biogen Provide Update on Phase 4 RESPOND trial of Spinraza
5-Year Results from the Nurture Spinraza Trial Published
Scholar Rock Provide Update on Phase 2 TOPAZ trial of Apitegromab
Novartis presents data on long-term treatment with Zolgensma
Taldefgrobep Alfa Receives FDA Fast Track Designation for SMA
ROCHE Community Update – SUNFISH Trial
NHS England Provides Further Guidance around Definition of Non-Success of Zolgensma
Results Published from STRONG Trial of Zolgensma
Conversations with health professionals – new webinar!
