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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…
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,…
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…
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…
CMT4 Research
…the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, for undertaking a CMT4A gene therapy project. Taysha and the CMTA will…
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…
Sorbitol Dehydrogenase (SORD) Deficiency
…of their specific disease. It also provides the opportunity to participate in a clinical trial of an investigational new treatment targeting the underlying cause of SORD Deficiency. If you suspect…
REPORT CONCEPTSPREADS72
…will continue to be moni- tored for one year after treatment. CMT4A mouse models have also been treated with the gene therapy and are currently being assessed to measure treatment…
2021_Spring_CMTA_Report
…depressing: Some great researchers were working on CMT, but because there were no pharmaceutical companies involved, the research wasn’t being translated to treatments. HOW DID WE GET FROM THERE TO…