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Rare Genetic Muscle Diseases
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Rare Genetic Muscle Diseases trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Rare Genetic Muscle Diseases trials you may qualify forThe purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an…
This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givinos…
The purpose of this research is (1) to identify disease specific walking-related digital biomarkers of disease severity, and (2) monitor longitudinal changes in…
The goal of this interventional study is to demonstrate the feasibility and tolerability of music and movement intervention for children with congenital DM1, wh…
The purpose of this study is to collect and study key medical data about several ultra-rare GSDs (Glycogen Storage Diseases) including, but not limited to, GSD…
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, i…
Over the past century, the progress in biomedical sciences gave outstanding contributions to the understanding of human conditions, and even curing some of them…
This phase II trial studies how well cabozantinib-s-malate works in treating younger patients with sarcomas, Wilms tumor, or other rare tumors that have come ba…