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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…
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…
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…
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…
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…
Keep Hope Alive for Everyone Living with CMT
…treatments are found to prevent/stop progression of CMT and that a cure will soon be within reach. But hope is more than just optimism: it is based on an expectation…
CMTA and Taysha Gene Therapies Announce Collaboration for CMT4A
…Gene Therapies (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS)…