Myotonic Dystrophy clinical trials at UCSD
2 research studies open to eligible people
Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
open to eligible people ages 16 years and up
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).
La Jolla, California and other locations
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
open to eligible people ages 18-70
Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and limited biological understanding of the phenotypic heterogeneity of Myotonic Dystrophy 1 by examining strategies to improve the reliability by making further refinements in our sample collection and analysis procedures by developing strategies for managing patient heterogeneity going forward. Funding Source- FDA OOPD
La Jolla, California and other locations
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