Organizations
There are several organizations that provide support and advocacy for rare therapies.
Support Organizations:
- National Organization for Rare Disorders
- Muscular Dystrophy Association
- Parent Project Muscular Dystrophy
- Cure SMA
- Spinal Muscular Atrophy Foundation
Government Organizations:
Clinical Team Video Insights
Our specialty care team—including nurses, patient care advocates, dietitians, and pharmacists—works together to support people living with rare conditions. Watch to learn how personalized, compassionate care helps guide patients every step of the way.