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Phase 3
A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Long-Term Clinical Benefit of Govorestat in Patients With Charcot-Marie-Tooth-Sorbitol Dehydrogenase Deficiency (CMT-SORD)
This study is designed to assess the long-term efficacy and clinical benefit of AT-007 in patients with CMT-SORD. This randomized, double-blind study will asse…
Charcot-Marie-Tooth Disease With Sorbitol Dehydrogenase Deficiency (CMT-SORD)
Applied Therapeutics, Inc.NCT07191912
Observational
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults withNeuromuscular Disease
This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adult…
Neuromuscular Diseases (NMD)Amyotrophic Lateral SclerosisMyasthenia Gravis+15 more
University of Missouri-ColumbiaNCT07478172
Observational
Lower Extremity Gait, Mobility and Balance Confidence in Charcot-Marie-Tooth Patients With Cavovarus Foot Deformity
The overall objective of the proposed research is to begin to better understand the potential benefits and limitations of ankle -foot orthosis (AFO) use in the…
Charcot Marie Tooth Disease (CMT)
Bopha ChreaNCT07188415
The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to s…
LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more