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CMTAreportSpring2023spreads
…CMTA Partner On ASO Treatment for CMT1A 2023 ‘Pivotal’ Year for PXT3003 Treatment For CMT1A as Manufacturing Shifts to U.S. CMTA partner Pharnext announced Jan. 26 that it has completed…
CMTAfall2022web
…virtual meeting June 7. Mackenzie shared valuable information on treatment goals for bracing, treatment options, who’s a candi- date, what to expect at a visit and much more. The branch…
CMTAreportSummer2022web
…quite strong compared to the beginning of treatment. I always used a handrail going up and down stairs before treatment, but now I walk up and down with some pace…
The Spring 2023 CMTA Report
…of interest: New CMT1X Mouse Model Patients as Partners in Research Tour of Conklin Lab Life-Changing Pharnext Moves Px3003 Production to Us ASO Treatment for CMT1A Circumnavigating the Globe New…
CMTA and ToolGen Announce CMT1A Collaboration
CMTA and ToolGen Announce Collaboration to Develop Potential Treatment for CMT1A Using Gene Therapy (Glenolden, PA, October 7, 2021) The Charcot–Marie–Tooth Association (CMTA), the largest philanthropic funder of CMT research…
Natural History of TRPV4 Neuromuscular Disease – CMT2C
…in animal models of this condition has shown that disease mutations cause TRPV4 to be overactive, and drugs that block the channel are effective as treatments in these models. This…
CMT Awareness Month 2022
…with the disease, raising awareness is imperative. The CMTA is a patient-led nonprofit that is aggressively searching out a treatment for CMT by putting together a group of top-flight researchers…
Diana Bharucha-Goebel, MD Joins the CMTA-STAR Advisory Board
…is also interested in better understanding how we efficiently and safely target the peripheral nervous system with viral mediated gene transfer treatment approaches, studying total vector genome dose, parallel preventative…
Finding Your CMT Gene
Finding Your CMT Gene – The Distant Cousin Project The effort to identify genes that cause rare diseases is more important than ever since effective treatments for genetically caused rare…
2021 STAR Accomplishments
…the body responds to a treatment for a disease) Initiatives in CMT1A, CMT1X, CMT2A, CMT1B and others in process: INC progress in developing muscle MRI and blood sample measurements of…