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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…
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 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…
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 Alliance Partner, Applied Therapeutics, Announces Positive 12-Month Results in its Ongoing INSPIRE Phase III Trial for CMT-SORD
…statement, Applied Therapeutics said the trial at its 12-month mark has shown a statistically significant correlation between sorbitol levels and the prespecified CMT Functional Outcomes Measure (CMT-FOM) and that treatment…
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…
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…
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…
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…