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What_Is_CMT_4-2017
…Stay Connected Get CMTA eNews/Update your lists: www.cmtausa.org/enews CMTA on Facebook: www.facebook.com/groups/CMTAssociation En Espanol: www.facebook.com/groups/CMTAespanol For Parents: www.facebook.com/groups/CMTAParents For Youth: www.facebook.com/groups/CMTAYouth CMTAthletes: www.facebook.com/groups/cmtathletes Webinars: www.cmtausa.org/webinars Patient/Family Conferences: www.cmtausa.org/pfcs Become a…
LetsBeatCMT Toolkit
…#LetsBeatCMT and educate the world about #CMT. Visit cmtausa.org for more information about CMT and join the fight. #CMTAFamily • Join the #CMTAFamily during #CMTAwareness Month & #LetsBeatCMT. Visit cmtausa.org…
What_Is_CMT_4-2017
…Stay Connected Get CMTA eNews/Update your lists: www.cmtausa.org/enews CMTA on Facebook: www.facebook.com/groups/CMTAssociation En Espanol: www.facebook.com/groups/CMTAespanol For Parents: www.facebook.com/groups/CMTAParents For Youth: www.facebook.com/groups/CMTAYouth CMTAthletes: www.facebook.com/groups/cmtathletes Webinars: www.cmtausa.org/webinars Patient/Family Conferences: www.cmtausa.org/pfcs Become a…
2022 Annual Report_spreads
…development for many types of CMT, including CMT1A, CMT1X, CMT2A, CMT2D, CMT2E, CMT2F, CMT2S, CMT4A and CMT4C, utilizing a spectrum of different technologies, including AAV delivery, gene silencing, gene replacement,…
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…
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…
Genetics-of-CMT
…CMT subtype Gene HNPP PMP22 (deletion) CMT1A PMP22 (duplication) CMT1B MPZ CMT1C LITAF/SIMPLE CMT1D EGR2 CMT1E PMP22 (point mutation) CMT1X GJB1 (Cx32) CMT2A MFN2 CMT2B RAB7 CMT2D GARS CMT2E NFL…
CMT_Fact_Sheet
…development of new drugs to treat CMT, to improve the quality of life for people with CMT, and, ultimately, to find a cure. Our vision is a world without CMT….
CMT Fact sheet_1-2020
…development of new drugs to treat CMT, to improve the quality of life for people with CMT, and, ultimately, to find a cure. Our vision is a world without CMT….