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CMT1A Research Breakthrough!
A paper published December 4th on the Journal of Clinical Investigation (JCI) website reveals an exciting potential treatment for patients with Charcot-Marie-Tooth (CMT), the progressive neuromuscular disease that affects more…
CMTA Board of Directors
…as leading the charge for development of treatments. Bernard Coulie Bernard is the CEO and President of Pliant Therapeutics, a NASDAQ-listed biopharmaceutical company focused on developing novel treatments for rare…
MDA and CMTA Fund Grant to Study Gene Therapy in Charcot-Marie-Tooth Disease
…whether treatment at later stages of the disease leads to improvements similar to those seen for treatment in the early stages. CMTA CEO Gilles Bouchard said, “Partnerships are at the…
Voice_of_the_Patient_Report_8-19-2019
…meeting, a parallel effort to FDA’s PFDD initiative to more systematically obtain patients’ perspectives on the burden of disease and impact of current treatments or lack thereof in Hyattsville, Maryland…
CMTA Report_Winter 2024 – STAR Special Edition_Download
…By supporting these efforts, you’re helping to turn groundbreaking research into real-world treatments that can change lives. SMALL MOLECULE THERAPIES: EXPANDING TREATMENT POSSIBILITIES Small molecule therapies offer a promising approach…
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…
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…
Understanding Charcot-Marie-Tooth Disease in Children
…no approved treatments for CMT. Understanding CMT in Children Children with CMT sometimes take longer to reach physical milestones, such as walking, hopping, jumping with two feet, or getting up…
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…