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Help the CMTA Develop Clinical Trials in a Dish
…(aka CMT1X/CMTX), GJB1 del mutation, male CMTX1 (aka CMT1X/CMTX), GJB1 (p.Ser26Leu) aka S26L, male CMT2A, MFN2 (p.His361Tyr) aka H361Y, male CMT2A, MFN2 (p.Arg364Trp) aka R364W, male CMT2A, MFN2 (p.Thr105Met) aka…
CMTA Alliance Partner, Applied Therapeutics, Announces Positive 12-Month Results in its Ongoing INSPIRE Phase III Trial for CMT-SORD
…statement, Applied Therapeutics said the trial at its 12-month mark has shown a statistically significant correlation between sorbitol levels and the prespecified CMT Functional Outcomes Measure (CMT-FOM) and that treatment…
CMT Type 4
…to Type 4 include: CMT4A – Ganglioside-induced differentiation-associated protein 1 (GDAP1) CMT4B1 – Myotubularin related protein 2 (MTMR2) CMT4B2 – SET binding factor 2 (SBF2/MTMR13) CMT4B3 – SET binding factor…
CMTA-Funded Research Database Identifies New Mutations
…evaluate CMT therapies. “The work in Züchner’s lab aligns perfectly with the CMTA’s Strategy to Accelerate Research (STAR),” CMTA CEO Amy Gray said. “Funding this project is another way the…
CMTA Announces Strategic Partnership with Acceleron to Advance CMT Treatment Options
…developing medicines to treat serious and rare diseases, including CMT. The CMTA’s Strategy to Accelerate Research (STAR) connects top CMT clinicians and academic researchers with pharmaceutical partners committed to developing…
2018_Winter_CMTA_Report
…Michelle Hayes National Branch and Community Relations Manager, michelle@cmtausa.org Leslie Nagel Marketing Coordinator, leslie@cmtausa.org Kim Magee Director of Finance and Administration, kim@cmtausa.org Jeana Sweeney Director of Community Engagement, jeana@cmtausa.org Kerry…
LER_Orthotic_Management_CMT PDF
…identifiable CMT: CMT1A, CMT1B, CMTX, and CMT2A.” According to Shy, CMT1A is considered the classic pheno- type. “These patients are likely to have been slow runners and clumsy as children,…
LER_Orthotic_Management_CMT
…identifiable CMT: CMT1A, CMT1B, CMTX, and CMT2A.” According to Shy, CMT1A is considered the classic pheno- type. “These patients are likely to have been slow runners and clumsy as children,…
Summer_2020_CMTA_Report
…Administration, kim@cmtausa.org Leslie Nagel Marketing Coordinator, leslie@cmtausa.org Laurel Richardson Director of Community Outreach, laurel@cmtausa.org Jeana Sweeney Director of Development, jeana@cmtausa.org INSIDE THIS ISSUE: RESEARCH New CMT Gene Identified……………………4 SPECIAL COVID-19…
2019_Spring_CMTA_Report
…your area? Contact CMTA Director of Community Outreach Laurel Richardson at laurel@cmtausa.org. 26 THE CMTA REPORT SPRING 2019 MELBOURNE, FL On November 17, 2018, the Melbourne, FL CMTA Branch had…