Cure SMA

Cure SMA is a nonprofit organization dedicated to advancing research, care, and advocacy for individuals affected by spinal muscular atrophy (SMA). It funds scientific research, supports families, and promotes access to new treatments for this genetic neuromuscular disease that historically was a leading genetic cause of infant mortality.

Background and Mission

Cure SMA began as a small grassroots network of families seeking a cure for SMA. Over time, it became a major international nonprofit connecting researchers, clinicians, and the SMA community. The organization’s central mission is to accelerate the development of effective therapies and, ultimately, a cure while ensuring people with SMA live full, supported lives.

Research and Medical Impact

The organization has invested over $80 million in SMA research, supporting projects from basic biology to clinical trials. It played a key role in advancing treatments such as nusinersen, onasemnogene abeparvovec, and risdiplam—all of which significantly changed the SMA treatment landscape. Cure SMA also manages the largest SMA patient registry, a critical tool for clinical research and therapeutic development.

Family and Community Support

Beyond research, Cure SMA provides practical and emotional assistance to affected families. Programs include newly diagnosed care packages, adaptive equipment, educational resources, and annual community conferences. These efforts help families navigate medical, social, and educational challenges associated with SMA.

Advocacy and Policy Work

Cure SMA collaborates with policymakers, industry, and healthcare providers to improve treatment access, newborn screening, and insurance coverage. Its advocacy has contributed to nationwide newborn screening for SMA in the United States, enabling earlier diagnosis and better outcomes.