Your legacy can help bring an end to CMT. Learn how a planned gift can make an impact for generations to come.
Medication Study
…lithium, and pyridoxine. Overall, Weimer and Podwall conclude that treatment with vincristine poses an “unacceptable risk to patients with known or possible CMT1A.” They further conclude that the use of…
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…
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…
Taysha Gene Therapies & CMTA Collaborate to Increase Genetic Testing Access for Giant Axonal Neuropathy (GAN)
…TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare…
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…
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…
CMTA Board of Directors
…as leading the charge for development of treatments. Bernard Coulie Bernard is the CEO and President of Pliant Therapeutics, a NASDAQ-listed biopharmaceutical company focused on developing novel treatments for rare…
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….