VCU registers END-EXT natural history study for myotonic dystrophy type 1; Dyne, Vertex collaborate
A new observational extension study on myotonic dystrophy type 1 progression is recruiting, sponsored by Virginia Commonwealth University with Dyne Therapeutics and Vertex Pharmaceuticals as collaborators.
This week's item covers a newly posted natural history study relevant to Dyne Therapeutics' myotonic dystrophy program.
- Virginia Commonwealth University is the lead sponsor of "Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension" (NCT07700225, acronym END-EXT), an observational study now recruiting, with Dyne Therapeutics, Vertex Pharmaceuticals Incorporated, and the Myotonic Dystrophy Clinical Research Network listed as collaborators. The study aims to characterize long-term disease progression over at least 4 years in at least 1,000 adults with myotonic dystrophy type 1 (DM1)1, and its cohort includes individuals moving over from the earlier parent trial, "Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1" (END-DM1; NCT03981575), along with participants enrolling for the first time1.
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