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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…
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…
CMTA-STAR Biotech & Alliance Partners
…endpoints to track treatment effects to be used in a planned gene therapy trial. Read more Frontage Laboratories Frontage Laboratories, Inc. is a global pharmaceutical development organization (PDO) advancing drug…
October 2023 Community Statement from Novartis
“The CMTA welcomes this community update from Novartis and applauds its leading role in the development of treatments for CMT. The CMTA has been maintaining regular contact, providing support and…
CMTA-STAR Clinical Expert Board Co-Chair Prof. Mary Reilly Honored with Prestigious MBE
…diagnosis, and treatment of Charcot-Marie-Tooth disease (CMT). As Co-Chair of CMTA-STAR’s Clinical Expert Board (CEB), she has played a vital role in shaping research strategies, accelerating therapeutic development, and improving…
CMTA Appoints Biotech Industry Veteran Suzanne Bruhn, PhD as New CEO
…treatments for rare diseases, will take the helm of the patient-led nonprofit as it moves to the next phase of its search for a cure for CMT. Dr. Bruhn brings…
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…
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…
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…