Programs and results
What we aim to solve
Our programs
What are the organization's current programs, how do they measure success, and who do the programs serve?
Therapeutic Research for ADSSL1 Myopathy
We are 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
Where we work
External reviews

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Financials
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Operations
The people, governance practices, and partners that make the organization tick.
Connect with nonprofit leaders
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Connect with nonprofit leaders
SubscribeBuild relationships with key people who manage and lead nonprofit organizations with GuideStar Pro. Try a low commitment monthly plan today.
- Analyze a variety of pre-calculated financial metrics
- Access beautifully interactive analysis and comparison tools
- Compare nonprofit financials to similar organizations
Want to see how you can enhance your nonprofit research and unlock more insights? Learn More about GuideStar Pro.
This organization has no recorded board members.