- Orphan Drug Designation for TSHA-120 for Treatment of GAN
Taysha 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 Navigator
The 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 Deficiency
Applied 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 Editing
CMTA 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 ... - CMTA-STAR Awards $354,826 for CMT Clinical Trial
CMTA-STAR Awards $354,826 for CMT Clinical Trial
(Glenolden, PA, December 7, 2021) The Charcot-Marie-Tooth Association Strategy to Accelerate Research (CMTA-STAR) awarded Dr. Mary M. Reilly and her team at the UCL Queen Square Institute of ...
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