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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…
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…
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,…
CMTA’s $300K Investment in CRISPR for CMT2A
…Current research suggests that silencing (turning off) only the mutated copy could restore peripheral nerve health, making gene editing an encouraging approach for treatment. Led by Bruce Conklin, MD, CMTA-STAR…
Accelerating Genetic Therapy for CMT4B1: A CMTA Research Update
…research for CMT4B1 is a critical step toward expanding treatment possibilities for the broader CMT community,” said Katherine Forsey, PhD, CMTA’s Chief Research Officer. “Through CMTA-STAR, we are not only…
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…