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description=Discover how CMTRF is accelerating treatments and cures for Charcot-Marie-Tooth disease. Learn about our targeted research pipeline and how you can help end CMT.;

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cmt research foundation changing the legacy of cmt cmt research foundation changing the legacy of cmt what is cmt about cmt type1 what is cmt1a what is cmt1b what is cmt1e what is hnpp type2 what is cmt2a type4 what is sord typex understanding cmt1x cmtx1 and gjb1 frequently asked questions watch our video series learn about treatments in development research research approach research projects we fund research pipeline gene therapy clinical trials apply for funding emerging researcher award news stories news cmt stories take action about us who we are board of directors research advisory council scientific advisory board research foundation staff events financials contact us velocity global cmt research convention donate cmt research foundation committed to a cure investing in innovative research to find treatments and cures for charcot marie tooth disease donate now research we fund clinical trials register today receive our newly diagnosed cmt patient package delivered to your home or via email request new patient package understand cmt as a recently diagnosed patient find therapies under development take action news latest cmt news explore more news project report a negative result points to the way forward in cmt4b aug 6 2026 cmtrf funded research a recent study found that a drug that helps other nerve diseases makes cmt4b1 worse and figuring out why has opened a more promising path toward treatment including a possible path forward for the related subtype cmt4b2 this work was funded jointly by the muscular read more cmt orgs come together to advocate for new clinical trial framework aug 5 2026 cmt research updates organizations focused on charcot marie tooth cmt disease have aligned with clinicians and pharmaceutical companies to share a set of recommendations for how cmt clinical trials should be designed with the recommendations published in journal of the peripheral read more the promise society a new way to support cmt research aug 4 2026 cmtrf foundation news there are moments when the future you ve been fighting for finally comes into view for the cmt research foundation this is one of those moments because of our community we are closer than ever to changing what it means to live with cmt the research we re funding read more register for the 6th annual global cmt research convention learn more cmt affects 1 in 2 500 people in the united states and more than 3 million people worldwide there is currently no cure for cmt donate now who we are accelerating a cure through research funding the cmt research foundation is a patient led non profit 100 focused on delivering treatments and cures for charcot marie tooth which affects 3 million people worldwide since 2018 our donors and scientists have put cmt front and center in the pharmaceutical biotech and investor communities making a cure for all types of cmt a not too distant reality we have funded 6 therapeutics that are in preclinical or active trials see full research pipeline featured content cmtrf case studies novartis ekd060 compound for cmt1a is going to clinical trials novartis acquired cmtrf funed partner dtx pharma in 2023 for 1 billion dtx pharma created a platform that delivers therapeutics to cells in a safe way we introduced dtx pharma to cmt and funded their first cmt research with a grant of 128 000 the results from the testing were positive and novarts is now taking it to clincial trials learn more cmtrf underwrites augustine therapeutics hdac6 inhibitor now the belgian pharmaceutical company is in a phase 1 clinical trial cmtrf helped augustine therapeutics underwrite the development of novel potent and subtype selective small molecule inhibitors of the cytosolic histone deacetylase 6 hdac6 enzyme to reverse and inhibit the pathophysiological changes associated with neuromuscular diseases now augustine therapeutics has completed their series a financing round and raised 85 million which will be used to advance their lead candidate agt 100216 through a phase i clinical trial in may 2025 augustine announced the dosing of their first patient in a phase 1 clinical trial learn more armatus bio licenses arm 101 from cmtrf funded partners dr kleopa cyprus institute of neurology and genetics and dr harper nationwide children s hospital in ohio for clinical trials drs kleopa and harper developed a therapy that safely delivers mirna a pmp22 silencer to the peripheral nervous system upon successful completion of their cmtrf funded project armatus bio licensed arm 101 for further testing and now the therapeutic treatment is preparing for clinical trials learn more featured content we bring you the latest discoveries innovative research and inspiring stories project report a negative result points to the way forward in cmt4b aug 6 2026 cmtrf funded research a recent study found that a drug that helps other nerve diseases makes cmt4b1 worse and figuring out why has opened read more cmt orgs come together to advocate for new clinical trial framework aug 5 2026 cmt research updates organizations focused on charcot marie tooth cmt disease have aligned with clinicians and pharmaceutical companies read more the promise society a new way to support cmt research aug 4 2026 cmtrf foundation news there are moments when the future you ve been fighting for finally comes into view for the cmt research foun read more deadline extended submit your abstract for the 2026 global cmt research convention jul 28 2026 cmtrf foundation news we re extending our abstract submission deadline for oral presentations the original oral presentation abs read more apply now emerging researcher forum at the 2026 global cmt research convention jul 28 2026 cmtrf foundation news the emerging researcher forum is a closed invite only session held the morning of day one of the 2026 global cmt read more the full agenda 2026 global cmt research convention jul 16 2026 cmtrf foundation news charcot marie tooth disease drug development is at a pivotal moment more active drug trials are underway than ever b read more webinar behind the scenes 2026 global cmt research convention preview jul 16 2026 cmtrf foundation news charcot marie tooth disease drug development is at a pivotal moment with more active drug trials underway than ever read more taking aim at cmt1j with a genetic medicine jul 16 2026 cmtrf funded research the cmt research foundation in partnership with the 1j foundation has funded a new study at the university of mia read more introducing the cmtrf journal club a new interactive series for cmt researchers jul 14 2026 cmt research updates the cmt research foundation is launching a new journal club webinar series designed specifically for researchers wo read more why you should be in the room dr anna greka to keynote global cmt research convention jul 13 2026 cmtrf foundation news don t miss friday s keynote at the global cmt research convention september 24 to 26 2026 cambridge read more help us find a cure donate now donate now help us find a cure newsletter signup stay up to date on new cmt research treatments and clinical trials address 4062 peachtree road suite a209 atlanta ga 30319 phone number 404 806 7180 email email protected follow follow follow follow follow 2026 cmt research foundation privacy policy manage consent to provide the best experiences we use technologies like cookies to store and or access device information consenting to these technologies will allow us to process data such as browsing behavior or unique ids on this site not consenting or withdrawing consent may adversely affect certain features and functions functional functional always active the technical storage or access is strictly necessary for the legitimate purpose of enabling the use of a specific service explicitly requested by the subscriber or user or for the sole purpose of carrying out the transmission of a communication over an electronic communications network preferences preferences the technical storage or access 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