Cure Adssl1

Cure ADSSL1 was established by Priyanka Kakkar and Naveen Baweja when both their children were diagnosed with an ultra-rare neuromuscular disease called ADSSL1 Myopathy. Cure ADSSL1 is a parent-run non-profit foundation with the mission to find a cure for ADSSL1 Myopathy. The impact of this disease on the patients is debilitating with loss of mobility in arms and legs, reduced function of heart, lungs, and swallowing muscles. Impact on patients includes decreased ability to perform activities of daily living and maintain functional independence. We are working with leading researchers and supporting research for two potential therapeutic options for ADSSL1 Myopathy: 1) Drug Repurposing 2) Gene Replacement Therapy