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Charcot-Marie-Tooth Type 1A Neuropathy
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Charcot-Marie-Tooth Type 1A Neuropathy trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Charcot-Marie-Tooth Type 1A Neuropathy trials may be worth asking aboutNorth America
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A Phase 1b/2a Clinical Trial to Evaluate the Safety and Efficacy of EN001 in Patients with Charcot-Marie-Tooth Disease type 1A(CMT1A) (Phase 1b: Open-label, Dos…
The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A.
This is a 2-year follow-up study of a cohort of 35 CMT1A patients and 20 healthy volunteers. The main objective is identifying prognostic markers for CMT1A usin…
The pathophysiological process common to neuropathies is fatty replacement of muscle tissue, more commonly known as intramuscular fat fraction (F.F). MRI is an…
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple…
This is a 2-year follow-up study of a cohort of 60 CMT1A patients. The objective is to identify markers allowing to better understand the phenotypic variability…