CureADSSL1

Cure ADSSL1 is a non-profit research foundation and patient advocacy organization dedicated to finding a cure for ADSSL1-deficient myopathy, a rare and progressive muscle-wasting disease. The foundation accelerates therapeutic development by funding high-impact research, including gene therapy and small molecule drug discovery programs. Cure ADSSL1 also focuses on essential clinical readiness activities, such as establishing a global patient registry and conducting natural history studies to identify disease biomarkers. By fostering collaborations between academic scientists, clinicians, and industry partners, the organization aims to bridge the gap between basic research and clinical application. Their ultimate goal is to provide life-changing treatments for individuals affected by this genetic neuromuscular disorder.