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Our Scientific Advisory Committee is now working to create alternative clinical trial designs, new biomarker criteria and more powerful statistical models that can be used for more efficient and predictable evaluation of treatments for very rare diseases. In turn, these models will be used to shape a new regulatory environment that will inspire confidence and new investment in rare disorders. Our direct costs in contracting with the experts needed, and conducting FDA Workshops to review the findings for these critical guidance documents, is projected at $250,000.

Your donation will help change the system and ultimately change the lives of the millions living with a rare disorder; by transforming the science we already have into treatments for patients like Matthew.