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Understanding Charcot-Marie-Tooth Disease in Children
…no approved treatments for CMT. Understanding CMT in Children Children with CMT sometimes take longer to reach physical milestones, such as walking, hopping, jumping with two feet, or getting up…
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…
Accelerating Genetic Therapy for CMT4B1: A CMTA Research Update
…research for CMT4B1 is a critical step toward expanding treatment possibilities for the broader CMT community,” said Katherine Forsey, PhD, CMTA’s Chief Research Officer. “Through CMTA-STAR, we are not only…
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…
Coronavirus (COVID-19) Update
…COVID-19 has now been detected in 60 locations internationally, including in the United States. The resources below provide information about the virus, how it spreads, symptoms, prevention/treatment and more. Please…
CMTA Fuels Breakthrough Imaging Research to Address CMT2A’s Cellular Weakness
…Accelerate Research (CMTA-STAR) project will equip scientists with tools to accelerate the development of potential treatments for CMT2A and other axonal forms of CMT. Investigating CMT2A at the Cellular Level…
New Gene Therapy Development Program for CMT2A
Philadelphia, PA – September 9, 2019 – Passage Bio, a genetic medicines company developing AAV-delivered gene therapies for the treatment of rare monogenic central nervous system (CNS) diseases, today announced…
Pharnext Announces Disappointing Results for CMT1A Drug Trial PXT-3003
…can be influenced by placebo and training effects, making it difficult to determine the impact of treatment. In particular, the company said, the Overall Neuropathy Limitation Scale (ONLS), which measures…