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Muscular Dystrophy Association and Charcot-Marie-Tooth Association Announce Collaborative Non-Viral Gene Therapy Research Grant
…cases are attributed to mutations in just four genes: PMP22 (CMT1A), MPZ (CMT1B), GJB1 (CMT1X), and MFN2 (CMT2A). CMT can be inherited in several ways: autosomal dominant (through a faulty…
CMTAM 2023 Toolkit
FallReportSpreads
2022 Annual Report_spreads
What_Is_CMT_4-2017
What_Is_CMT_4-2017
LetsBeatCMT Toolkit
Applied Therapeutics Announces Positive Sorbitol Reduction Data From the Ongoing Phase 3 INSPIRE Trial in Sorbitol Dehydrogenase (SORD) Deficiency
…evaluating the effect of once-daily oral AT-007 in approximately 50 patients age 16-55 with SORD Deficiency in the US and Europe. SORD Deficiency (also called SORD Neuropathy or CMT-SORD) is…
Sperm Donor with Gene for Charcot-Marie-Tooth Disease Passes It on to Multiple Families
…using Donor X’s sperm. Both have a rare form of CMT—Dominant Intermediate B, a group of CMT subtypes in which nerve conduction does not comport with either axonal CMT or…
Awareness Month: A STAR Researcher’s Perspective
…ten years that we’ve been trying to develop gene therapy initially for the x-linked type of CMT and, more recently, also for CMT4C, CMT1A and CMT2A. We work very hard…