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MDF Research Publications

MDF Publishes Article in Therapeutic Innovation and Regulatory Science

The first publication generated by the Myotonic Dystrophy Foundation, titled "Patient-Centered Therapy Development for Myotonic Dystrophy: Report of the Myotonic Dystrophy Foundation–Sponsored Workshop (pdf)," was published in Therapeutic Innovation and Regulatory Science. The report examines the status of biomarker, endpoint and clinical trial design for myotonic dystrophy therapy development, and lays the groundwork for the inclusion of the patient and caregiver voice in DM therapy development. It also includes a report on the Benefits and Risks Study MDF conducted with Silicon Valley Research Group.

MDF Releases Voice of the Patient Report on the Myotonic Dystrophy Patient-Focused Drug Development Externally-Led Meeting

The report summarizes patient and caregiver input on the disease burden of myotonic dystrophy types 1 and 2, and what specific symptoms most impact their daily lives. It also provides patient and caregiver perspectives on what clinically meaningful benefits would be for future DM therapies. Access the DM Voice of the Patient Report and the FDA submission letter. Watch videos of the meeting here and here.





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