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Charcot-Marie-Tooth Disease
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Charcot-Marie-Tooth Disease trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
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Background: Pyrimidine and purine metabolism disorders (DPPMs) affect how the body metabolizes chemicals called pyrimidines and purines. DPPMs can cause dysfun…
This is a prospective longitudinal natural history study with a retrospective cross-sectional arm aimed at determining the natural history of sphingosine phosph…
Charcot-Marie-Tooth (CMT) disease is caused by a genetic abnormality involving the PMP22 gene, resulting in demyelination of the peripheral nerves. Demyelinatio…
The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A.
Down syndrome (DS) is associated with cognitive deficits, caused by alterations in neuroplasticity and synaptic transmission. Non-invasive brain stimulation tec…
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…
The purpose of this study is to assess the prognostic value of absolute value and kinetic of neurofilament light chain (NFL) collected at different time points…
Carpal tunnel syndrome is a common condition caused by compression of the median nerve at the wrist. It can result in pain, numbness, tingling, weakness, and di…