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Dystrophia Myotonica 1
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Dystrophia Myotonica 1 trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Dystrophia Myotonica 1 trials you may qualify forThe 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)…
This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 year…
The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PG…
The goal of this observational study, conducted in participants' homes and requiring no travel to a study site, is to better understand disease variability in p…
The purpose of this study is to learn about the long-term safety and tolerability of PGN-EDODM1 in participants with myotonic dystrophy type 1 (DM1) who have co…
The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myo…
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-de…
This cross-sectional observational study aims to determine the frequency of falls in patients with myotonic dystrophy type 1 and to identify factors affecting d…
This is a prospective multicentric Italian study to evaluate the arrhythmic risk in myotonic dystrophy type 1.