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Dystrophia Myotonica 1
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Dystrophia Myotonica 1 trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
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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)…
The goal of this study is to investigate the effects of personalized exercise treatment on dynamic balance and physical function in comparison with regular foll…
Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history;…
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in par…
Myotonic dystrophy type 1 (DM1) can affect people in many different ways, even in the same family. The symptoms that children experience can be different and mo…
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 ability of an individual to conceive some alternative representations and to behave in a flexible manner would emerge from preschool age and drastically imp…
Myotonic dystrophy type 1 (DM1) is an autosomal dominant multisystem disorder caused by an expanded CTG trinucleotide repeat in the 3' untranslated region of th…