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CMTA-STAR Alliance Partner Actio Biosciences Receives FDA Orphan Drug Designation and Rare Pediatric Disease Designation for Their Treatment in Development for CMT2C
…urgent need for new treatments for these patients.” We celebrate this encouraging news from Actio Biosciences and look forward to the continued progress in bringing effective treatments to those affected…
CMTA’s $450K Investment Targets New Approach to Treatment for Patients with CMT1A and CMT1B
The Charcot-Marie-Tooth Association (CMTA), the largest philanthropic funder of Charcot-Marie-Tooth (CMT) disease research aimed at bringing treatments to patients, announced today an investment of $450K into a new scientific approach…
Shift Pharmaceuticals, CMTA Partner on ASO Treatment for CMT1A
Shift Pharmaceuticals, CMTA Partner on ASO Treatment for CMT1A GLENOLDEN, PENNSYLVANIA, USA, NOVEMBER 3, 2022 — Shift Pharmaceuticals, a privately held company that is developing antisense oligonucleotides (ASOs) to treat…
Consensus_Statement_on_Surgical_Treatment_of_CMT
…the success or fail- ure of their treatment? A history of genetic testing should be noted. Although not required for surgical treatment, the CMT genotype may be helpful in better…
Data Shows Benefit with PXT3003 in Patients with CMT1A
…treatments. Pharnext has two lead products in clinical development. PXT3003 completed an international Phase III trial with encouraging topline results for the treatment of Charcot-Marie-Tooth disease type 1A (‘CMT1A’) and…
CMT1A Ionis Press Release
New Research Shows Promise for CMT Progressive Neuromuscular Disease Potential treatment offers hope for over 2.8 million affected worldwide Glenolden PA, December 4, 2017—A new paper published December 4th on…
A Cure for Caroline and Other CMT Stories
Caroline’s journey with a rare and serious form of CMT began when she was just 9 months old. Today, a cure for Caroline may open the door to treatments and…
Webinar Recordings
…spent the last decade specializing in treatment of adults with acquired neurological impairments. She is certified in neuro-developmental treatment and the use of physical agent modalities. She has specialty training…
What’s New in CMT Research and Care? A 2025 Update Published by CMTA-STAR Advisory Board Member Davide Pareyson, MD
…proper care and treatment planning. Visit CMTA’s Diagnosing CMT page to learn more. Comprehensive Care: Managing CMT Today While there are no FDA-approved treatments for CMT, CMT specialists continue refining…
Applied Therapeutics Announces Positive Sorbitol Reduction Data From the Ongoing Phase 3 INSPIRE Trial in Sorbitol Dehydrogenase (SORD) Deficiency
…a pre-specified interim analysis of the ongoing Phase 3 INSPIRE trial, AT-007 reduced sorbitol levels by a mean of approximately 52% (or approximately 16,000ng/ml) over 90 days of treatment (p…