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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….
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…
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…
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…
CMTA Report_Winter 2024 – STAR Special Edition_Download
…By supporting these efforts, you’re helping to turn groundbreaking research into real-world treatments that can change lives. SMALL MOLECULE THERAPIES: EXPANDING TREATMENT POSSIBILITIES Small molecule therapies offer a promising approach…
CMTA’s $300K Investment in CRISPR for CMT2A
…Current research suggests that silencing (turning off) only the mutated copy could restore peripheral nerve health, making gene editing an encouraging approach for treatment. Led by Bruce Conklin, MD, CMTA-STAR…
CMTA Event Schedule Update from CEO Amy Gray
…prevention/treatment and more. We ask you to review the information contained in these links to be better informed about the coronavirus. CDC: Prevention & Treatment World Health Organization: Coronavirus Disease…
CMTA Awards $225,483 for Study On Using ASOs to Treat CMT2E
…a functional protein. They are short man-made pieces of genetic material designed to bind to a specific target messenger, RNA (mRNA). The Food and Drug Administration has approved ASO-based treatments…