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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.

5-Year Results from the Nurture Spinraza Trial Published

Treatments & Research
News

Scholar Rock Provide Update on Phase 2 TOPAZ trial of Apitegromab

Treatments & Research
News

Novartis presents data on long-term treatment with Zolgensma

Information
News
Treatments & Research

Taldefgrobep Alfa Receives FDA Fast Track Designation for SMA

Information
Treatments & Research

ROCHE Community Update – SUNFISH Trial

Information
News
Treatments & Research

NHS England Provides Further Guidance around Definition of Non-Success of Zolgensma

Treatments & Research
Information
News

Results Published from STRONG Trial of Zolgensma

Information
News
Treatments & Research

Conversations with health professionals – new webinar!

Information
Treatments & Research

Interim Results from Risdiplam Jewelfish Trial Published

Information
Treatments & Research
test tubes in a science laboratory.

NHS England agrees to allow access to Sapphire Trial to enhance treatments for SMA

Information
Treatments & Research