FDA Approves First Muscle-Targeted Therapy for SMA

Innovations in Care

We provide programs and services that transform the lives of people affected by neuromuscular disease.

Sparkle Trial Awareness Webinar

August 13th, 2026
On-Demand Webinar

The Sparkle Study is a clinical research study testing a possible new (investigational) medicine for children and teenagers with SMA.

Watch On Demand


Thank you to our supporter:

Argenx