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Research in Charcot–Marie–Tooth Disease Type 1A (CMT1A)
CMT1A Research Since 2008, the CMTA-STAR has made historic progress in developing a treatment for CMT1A. The chart above shows the major milestones we have reached. View the projects currently…
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…
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…
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…
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 1 CMT Gene Therapy Project Now Underway
…CMT (type 1 and most type 4).” “Gene therapy continues to show promise for the treatment of neuromuscular disease,” said Amanda Haidet-Phillips, Ph.D., one of the MDA’s scientific portfolio directors….
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…
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…
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….