- Diana Bharucha-Goebel, MD Joins the CMTA-STAR Advisory Board
In a suite of exciting appointments, the Charcot-Marie-Tooth Association (CMTA) Strategy to Accelerate Research (STAR) Advisory Board is delighted to announce the appointment of Dr. Diana Bharucha-Goebel to their Clinical Expert Board (CEB). The CEB ...
Read more ... - Data Shows Benefit with PXT3003 in Patients with CMT1A
New 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 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 ...
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