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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…
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…
CMTA-STAR Research for Axonal Forms of CMT
…to the market in the near future. The CMTA’s funding and operations focus is on translational research that will lead as directly as possible to therapeutic treatments for CMT. Axonal…
Innervator Hall of Fame
…invest in invaluable educational initiatives and treatment-focused research. Join our action-oriented game changers by becoming a CMTA INNERVATOR. Tracy Adamson Richard Arsenault Aaron Baker David Balbi Kelly Banner James Barker…
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…
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…
Sperm Donor with Gene for Charcot-Marie-Tooth Disease Passes It on to Multiple Families
…treatment for the painful and debilitating symptoms that CMT causes.” In a happy footnote to the Erica’s story, Frankie was recently accepted to Camp Footprint, the country’s only sleepaway camp…
The Summer 2023 CMTA Report
…of interest: CMT Patients Urge Faster Clinical Trials Applied Therapeutics Research Update DTx Treatment for CMT1A Under the Microscope: ASOs CMTA Funds Preclinical Work on CMT1B Mice Summer with CMT…
A New Therapeutic Strategy for CMT2D
…for nerve cell survival. Through experiments that identified the cause of this transport disruption, the scientists were able to develop a treatment strategy that improves this critical transportation system in…
CMT Type 2
…the function of the working protein and somehow treatment will need to silence that abnormal copy or cut it out. If it can be silenced, blocked or cut it out…