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Your Participation Matters: Join the CMT2S Research Study
CMT2S: A Potential New Treatment on the Horizon Charcot-Marie-Tooth Disease Type 2S (CMT2S) is a severe axonal type of CMT caused by certain autosomal recessive mutations of the IGHMBP2 gene….
CMT2A Research
…reagents can prevent disease pathology in a rodent model of CMT2A. PROJECT GOAL: EARLY PHASE TREATMENT TRIALS OF CMT2A Grant Amount: $18,942 Principal Investigator: Michael Shy, MD, University of Iowa…
CMTA-STAR Advisory Board Member Dr. D’Antonio Publishes in Brain, Identifies Potential Therapeutic Target for CMT1B
…supporting proper myelination. This finding offers a potential pathway for future treatment approaches. “Our research shows that XBP1 plays a key role in helping Schwann cells manage cellular stress and…
Medications and Charcot-Marie-Tooth Disease
There currently are no available treatments that act to slow the progression of Charcot-Marie-Tooth disease. There are promising CMT research studies that indicate that this may change in the near…
Hereditary Sensory Neuropathy Serine trial (SENSE trial)
…variable weakness in the upper and lower extremities, for which there is no current treatment. Dr. Reilly’s team at UCL Queen Square Institute of Neurology plan to perform a 12-month…
MDA and CMTA Fund Grant to Study Gene Therapy in Charcot-Marie-Tooth Disease
…whether treatment at later stages of the disease leads to improvements similar to those seen for treatment in the early stages. CMTA CEO Gilles Bouchard said, “Partnerships are at the…
Type 2 Update: One Patient’s Diagnostic Odyssey with SORD
…was surprised to read about potential treatments. There are drugs currently in clinical trials that could jump-start the afflicted enzymes, effectively preventing further damage. I don’t think I’ve completely wrapped…
Coronavirus (COVID-19) and CMT
…impacts will the vaccine have on possible future gene therapy treatments or medications? Whether the vaccine will have an adverse impact on any course of treatment that you or your…
Coronavirus (COVID-19) and CMT
…impacts will the vaccine have on possible future gene therapy treatments or medications? Whether the vaccine will have an adverse impact on any course of treatment that you or your…
Acceleron Announces Topline Results from the Phase 2 Trial of ACE-083 in Patients with Charcot-Marie-Tooth Disease
…strength, function, quality of life, and safety over a 6-month primary treatment period, followed by a 6-month open-label treatment period. For additional information about this clinical trial, please visit www.clinicaltrials.gov….