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CMTAfall2022web
…risks of not achieving expected outcomes or lacking the statistical power to obtain meaningful results. Ongoing biomarker studies funded by the CMTA (for CMT1B, CMT1X, CMT2A, and CMT2F) depend on…
From Gene Discovery to Gene Editing: Closing the Diagnostic Gap and Developing CRISPR/Cas9 Capabilities in CMT
…work has led to the discovery of more than 25 genes in recent years, including the CADM3 (CMT2GG), COQ7 (dHMN-COQ7), ITPR3 (CMT1J), and SORD (CMT-SORD) genes. This program has a…
2019_Summer_CMTA_Report
…laurel@cmtausa.org Jeana Sweeney Director of Development, jeana@cmtausa.org INSIDE THIS ISSUE RESEARCH CMT4C—Gene Therapy …………………………………………………………………………6 Wearable Biomarkers …………………………………………………………………………..6 CMT COMMUNITY The Doctors of the CMTA ……………………………………………………………………….8 Why We Give…………………………………………………………………………………..14 FUN AND…
Type 1 CMT Gene Therapy Project Now Underway
…their behalf. To learn more about the CMTA, please visit www.cmtausa.org. Media contacts: Mary Fiance Director, Public Relations and Communications, MDA (646) 992-2908 mfiance@mdausa.org Marcia Semmes Media Relations, CMTA marcia@cmtausa.org…
CMTA Updates List of Neurotoxic Meds Potentially More Hazardous to CMT Patients
…Systematic Review. J Peripher Nerv Syst. 2023 May 30. doi: 10.1111/jns.12566. Epub ahead of print. PMID: 37249082. About the CMTA The CMTA is the leading patient advocacy organization driving CMT…
Type 1 CMT Gene Therapy Project Now Underway
…their behalf. To learn more about the CMTA, please visit www.cmtausa.org. Media contacts: Mary Fiance Director, Public Relations and Communications, MDA (646) 992-2908 mfiance@mdausa.org Marcia Semmes Media Relations, CMTA marcia@cmtausa.org…
CMT1X Research
…therapy for not only CMT1X but also other demyelinating forms of CMT (CMT1A and CMT4C). The CMTA convened a workshop with some of the world’s top gene therapy experts to…
2019_Summer_CMTA_Report
…laurel@cmtausa.org Jeana Sweeney Director of Development, jeana@cmtausa.org INSIDE THIS ISSUE RESEARCH CMT4C—Gene Therapy …………………………………………………………………………6 Wearable Biomarkers …………………………………………………………………………..6 CMT COMMUNITY The Doctors of the CMTA ……………………………………………………………………….8 Why We Give…………………………………………………………………………………..14 FUN AND…
CMT Subtype Biomarkers and Outcome Measures
…this study includes: 2 questionnaires (the CMT Health Index and Patient Global Impression of Change (PGIC)) that explore how you think CMT affects you CMT Functional Outcome Measure (CMT-FOM) which…
The_2018_CMTA_Report_35th_Anniversary_Edition
…Co-Chair, Clinical Expert Board FLASHBACK 1987 The Second International Conference at Columbia University was groundbreaking for the CMTA and the CMT community. mailto:andi@cmtausa.org mailto:frank@cmtausa.org mailto:leslie@cmtausa.org mailto:kim@cmtausa.org mailto:laurel@cmtausa.org mailto:jeana@cmtausa.org https://www.cmtausa.org mailto:info@cmtausa.org…