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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
Feel free to tag CMTA in your posts using the profiles below: Facebook: @CMTAssociation Twitter, Instagram, and TikTok: @CMTAUSA LinkedIn: @CharcotMarieToothAssociation Hashtags: #CMTAM23, #CMTAUSA, #CMTASTAR, #SeeCMT, #NavigatingCMT CMTA has created…
FallReportSpreads
…quality of life for those living with CMT. For more information about CMT Awareness Month 2023 and how to participate, visit the official CMTA website: https://www.cmtausa.org/get-involved/ cmt-awareness-month-2023/ THERE’S STILL TIME…
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…
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…
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,…
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…
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…
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…