- Data Shows Benefit with PXT3003 in Patients with CMT1ANew Data from the Open-Label PLEO-CMT-FU Trial Shows Sustained Benefit with PXT3003 in Patients with Charcot-Marie-Tooth Disease Type 1A After 5 Years of Total Trial Time New data from the ongoing Open-Label Phase III Extension Study ...Read more ...
- Orphan Drug Designation for TSHA-120 for Treatment of GANTaysha Gene Therapies Receives Orphan Drug Designation from the European Commission for TSHA-120 for the Treatment of Giant Axon Neuropathy (GAN) Clinical efficacy data for TSHA-120 provide quantitative evidence of long-term durability across all therapeutic dose ...Read more ...
- The CMTA Earns a Perfect Rating From Charity NavigatorThe Charcot-Marie-Tooth Association Earns a Perfect Rating From Charity Navigator GLENOLDEN, Pa., Feb. 23, 2022 (NEWSWIRE) — The Charcot-Marie-Tooth Association (CMTA), a 501(c)(3) nonprofit organization dedicated to the research and development of treatments for CMT, announces ...Read more ...
- Initiation of Registrational Phase 2/3 Study of AT-007 in SORD DeficiencyApplied Therapeutics Announces Initiation of Registrational Phase 2/3 Study of AT-007 in SORD Deficiency New York, December 16, 2021 (GLOBE NEWSWIRE) — Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline ...Read more ...
- CMTA Seed Money Draws NIH Support For Type 2 Gene EditingCMTA Seed Money Draws NIH Support For Type 2 Gene Editing, Neurofilament Projects (Glenolden, PA, December 14, 2021) The Charcot-Marie-Tooth Association Strategy to Accelerate Research (CMTA-STAR) today announced that the National Institutes of Health (NIH) awarded ...Read more ...
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