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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…
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…
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…
Voice_of_the_Patient_Report_8-19-2019
CMTA Report_Winter 2024 – STAR Special Edition_Download
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…
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,…
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…