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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…
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…
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…
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
2019_Summer_CMTA_Report
Gene Replacement Therapy in CMT4 Subtypes
…using a model replicating CMT4A. If successful, this approach could serve as a template for treating other types of CMT, such as CMT4B1 and CMT4D. “Recent advances in developing desirable…
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…