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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…
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…
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…
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…
CMTA’s CRISPR Research Initiative Advances for CMT1A and CMT1B
…CMTA’s global leadership in accelerating innovative research to bring treatments to CMT. Led by John Svaren, PhD, at the University of Wisconsin, this research is part of a larger initiative…
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…
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…