Your legacy can help bring an end to CMT. Learn how a planned gift can make an impact for generations to come.
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…
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….
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….
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…
Actio Biosciences Doses First Participant in Phase I Clinical Trial | Future Phase Ib Clinical Trial to Focus on CMT2C
…complex form of CMT that presents significant challenges for patients and their families. We proudly support Actio Biosciences in advancing this potential treatment.” The Phase I study is a randomized,…
CMTA Hails Novartis Acquisition of DTx Pharma And Its CMT1A Therapeutic Program
…of novel CMT treatments capable of reaching the peripheral nervous system. Under the terms of the agreement, Novartis will make an upfront payment of $500 million and additional payments of…
Studying Neurodegeneration in CMT1X Mouse Model
…If differences are found, the team can then investigate if those can be restored to wild type mice levels after treatment. At the in-life-phenotyping level, the team found the speed…
Bruce R Conklin, MD Joins the CMTA STAR Advisory Board
…use these model cells to test the effects of the CRISPR gene editing treatments they are developing. If the treatment is found to be effective in the human model cells,…
CMTA and Applied Therapeutics Announce Collaboration for CMT-SORD
…Inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Galactosemia, SORD Deficiency and PMM2-CDG. The Company is also developing AT-001, a novel potent ARI, for the treatment of…