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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 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….
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…
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….
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…
CMT1A Research Breakthrough!
A paper published December 4th on the Journal of Clinical Investigation (JCI) website reveals an exciting potential treatment for patients with Charcot-Marie-Tooth (CMT), the progressive neuromuscular disease that affects more…
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…
Voice_of_the_Patient_Report_8-19-2019
…meeting, a parallel effort to FDA’s PFDD initiative to more systematically obtain patients’ perspectives on the burden of disease and impact of current treatments or lack thereof in Hyattsville, Maryland…