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Charcot-Marie-Tooth Disease, Type 1
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Charcot-Marie-Tooth Disease, Type 1 trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Charcot-Marie-Tooth Disease, Type 1 trials you may qualify forThe project aims to perform both conventional nerve-conduction studies and axonal-excitability assessments using the TRONDF protocol in patients with selected f…
The Peroxisome Biogenesis Disorders (PBD) are a group of inherited disorders due to defects in peroxisome assembly causing complex developmental and metabolic s…
Background: Pyrimidine and purine metabolism disorders (DPPMs) affect how the body metabolizes chemicals called pyrimidines and purines. DPPMs can cause dysfun…
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or me…
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…
The objective of the ActiLiège Next study is to collect longitudinal data from patients and control subjects using a wearable magneto-inertial device. By collec…
First-in-human Phase 1/2, open-label study to evaluate the safety, tolerability, and efficacy of a single lumbar intrathecal dose of ELP-02 to individuals with…
Charcot-Marie-Tooth disease (CMT) is a hereditary peripheral nerve disease that causes causes muscle atrophy, muscle weakness, sensory loss, balance disorder, g…