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2021_Spring_CMTA_Report
…CMT2F, CMT2K, CMT4A and CMT4C, utilizing a number of different technologies, including AAV delivery, gene silencing, gene replacement and genome editing using CRISPR-Cas9 and antisense oligonucleotides (ASOs). The CMTA continues…
CMTA-STAR Research for Axonal Forms of CMT
…in-depth conversation about STAR please reach out to Jeana Sweeney, Chief Engagement and Gift Officer: jeana@cmtausa.org. Support CMT Research READ MORE ABOUT CMTA-STAR RESEARCH FOR AXONAL TYPES OF CMT CMT…
NMD Pharma Receives FDA Approval to Initiate Phase II Clinical Trial of NMD670 in CMT Patients
…clinical trial for their investigational drug, NMD670, in patients with Charcot-Marie-Tooth disease (CMT). This trial, named SYNAPSE-CMT, marks a significant milestone in the journey towards finding effective treatments for CMT….
CMT2A Research
CMT2A CMT2A is caused by dominant mutations in Mitofusin 2 (MFN2). The STAR team has developed two excellent rat models for CMT2A which are being made available to the research…
CMTA Report Fall 2021
…in The CMTA Report pertains to you. The CMTA assumes no liability for any information in The CMTA Report. ISSN #1067-0181 Vol. 36, No. 2 Email the CMTA at info@cmtausa.org…
2021_Winter_CMTA_Report
…Community Outreach, laurel@cmtausa.org Jeana Sweeney Director of Development, jeana@cmtausa.org INSIDE THIS ISSUE: RESEARCH Board Approves Grants for Inhibitor Work ………….7 CMT STORIES Filmmaker Doesn’t Let CMT Stop Her ………………….6 Educator…
CMTA Volunteer Spotlight
…laurel@cmtausa.org. CMTActive on Facebook (www.facebook.com/groups/cmtactive/) Benjy Hershorn (CMTActive Moderator and Houston Branch Leader) My name is Benjy Hershorn. Although born and raised in Kansas, I got to Texas “as fast…
A Cure for Caroline and Other CMT Stories
…Scarlett was diagnosed with a rare form of CMT called CMT2S. About CMT2S CMT type 2S is a newly discovered form of CMT caused by defects in the same gene…
Charcot–Marie–Tooth Disease and Breathing Problems
Does CMT cause Breathing Problems? Yes, CMT can cause breathing problems for some. For reasons not understood, not all individuals who have CMT will develop breathing problems. When breathing is…
X-linked CMT
…CMT1X/X1 is the second most common CMT subtype, accounting for approximately 17% of all CMT cases, second only to CMT1A, which accounts for approximately 60% of all CMT cases. The…