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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…
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…
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’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…
Nervosave: Shaping the Future of Genetic Therapy for CMT1A
…their condition and feelings toward this type of treatment. Why Participate in the Nervosave Survey? By participating in this survey, you contribute to research that could influence the future of…
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…
Actio Biosciences Doses First Participant in Phase I Clinical Trial | Future Phase Ib Clinical Trial to Focus on CMT2C
…complex form of CMT that presents significant challenges for patients and their families. We proudly support Actio Biosciences in advancing this potential treatment.” The Phase I study is a randomized,…
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…
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…