Cure CMD's 5-Year Research Goals


Cure CMD has achieved significant impact in its first nine years as the only nonprofit organization in the U.S. dedicated specifically to advancing research for the Congenital Muscular Dystrophies (CMDs) and improving the quality of life for affected individuals and their family members.

The Eugene Washington Engagement Award received through the Patient-Centered Outcomes Research Institute (PCORI) was fundamental in bringing experts together to both recognize the tremendous progress in research made since Cure CMD’s inception, and revitalize momentum toward clinical trials. The Award has reinvigorated and/or forged new collaborations and plans that will be instrumental in making tangible progress in understanding each of the five CMD subtypes and in developing therapeutic and care solutions for affected families.

As a product of the five- conference series made possible by this Award, Cure CMD constructed a “bench-to-bedside pipeline” (annex 1), which will be helpful in tracking progress and detecting areas that need particular attention to attain the goal. Furthermore, the conference series allowed us to unite all stakeholder voices: patients, caregivers, scientist, clinicians, pharmaceutical companies, and regulatory officials, resulting in a comprehensive list of critical elements and remaining questions that need action in pursuit of Cure CMD’s mission. Thus, with significant momentum built from the conference series funded in part by PCORI, Cure CMD proposes the following one-, three-, and five-year goals, which have been drafted with the input and support of top CMD experts and the patient community --> Go to Research Strategy

For more information on Cure CMD research, please reach out to Dr. Gustavo Dziewczapolski, Cure CMD's Scientific Director.


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Cure CMD is U.S. registered 501(c)(3) non-profit organization.
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