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Congenital Myotonic Dystrophy
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Congenital Myotonic Dystrophy trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Congenital Myotonic Dystrophy trials may be worth asking aboutNorth America
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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 overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and develop biomarkers…
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…
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…
This is a non-interventional, prospective, observational, multicentre study to evaluate the long-term safety and effectiveness of Namuscla in adult patients wit…