Resources for Study & Trial Participants

The Christopher Project Report: Report to the Myotonic Dystrophy Community: In order to better understand their needs, the Christopher Project asked patients with myotonic dystrophy and their family members/caregivers to report directly on their own experiences, from their own perspective, as experts in their own right. This report complements existing research and offers important, new insight into the myotonic dystrophy experience. Download the Christopher Project Overview and Highlights presentation. (.PPTX) Click here to watch the 2022 Ask-the-Expert webinar featuring key findings from the Christopher Project.

MDF Publishes Article in Therapeutic Innovation and Regulatory Science - This publication generated by MDF, titled "Patient-Centered Therapy Development for Myotonic Dystrophy: Report of MDF–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 - This 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.

Healthcare resource utilization, total costs, and comorbidities among patients with myotonic dystrophy using U.S. insurance claims data from 2012 to 2019. This study interrogated health insurance claims data to explore the clinical experience, healthcare resource utilization (HCRU), and all-cause costs for DM.