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Rare Genetic Muscle Diseases
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Rare Genetic Muscle Diseases trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Rare Genetic Muscle Diseases trials may be worth asking aboutNorth America
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Trials may have sites in multiple countries. Filtering shows any trial with at least one site in the selected country.
The 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…
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…
Research into novel therapies for rare, immune-mediated inflammatory diseases (IMIDs) is limited due to small patient populations. Patients with Behçet's diseas…
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…
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…
Over the past century, the progress in biomedical sciences gave outstanding contributions to the understanding of human conditions, and even curing some of them…