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Giant Axonal Neuropathy (GAN)
…removed and examined to look for swollen axons). A definitive diagnosis using genetic testing is available on a research basis only. What research is being done around GAN? Charcot-Marie-Tooth Association,…
2021 STAR Accomplishments
With our community’s help, the CMTA has invested over $17.5 million in CMTA-STAR research since 2008, making us the largest charitable funder of CMT research globally. Through the support of…
Initiation of Registrational Phase 2/3 Study of AT-007 in SORD Deficiency
…the CMTA’s research efforts are consolidated under the banner of STAR—which brings together the world’s largest network of biotech research partners, research scientists, clinicians and patients—and funds more CMT grants…
Type 2 Update: One Patient’s Diagnostic Odyssey with SORD
…scholar from University College of London— discovered the SORD mutation “hidden” from the gene analysis software that most researchers use. Using a different strategy, the researchers found the relevant variations…
Types of CMT
…because, by nerve conduction characteristics, it is neither demyelinating nor axonal – it is somewhere in between – it is intermediate. Learn more about how CMTA is accelerating research for…
From Gene Discovery to Gene Editing: Closing the Diagnostic Gap and Developing CRISPR/Cas9 Capabilities in CMT
…with the Inherited Neuropathies Consortium (INC), researchers at the University of Miami, led by CMTA Strategy To Accelerate Research (STAR) Board member Stephan Zuchner, MD, PhD, are working to solve…
What is CMT?
…Accelerate Research (STAR) in 2008, CMTA has been working with preeminent researchers, clinicians, patients and institutions in the global inherited neuropathy community to capitalize on breakthroughs in genetics. Unlike other…
CMTAreportSummer2023spreads
…SUMMER 2023 RESEARCH CMT PATIENTS URGE FASTER CLINICAL TRIALS ……………………………………………….. 10 APPLIED THERAPEUTICS RESEARCH UPDATE …………………………………………………… 11 DTx TREATMENT FOR CMT1A ……………………………………………………………………………….. 11 UNDER THE MICROSCOPE: ASOs ……………………………………………………………………….. 12 CMTA…
CMTA_2019_Form_990
…CHARCOT-MARIE-TOOTH DISEASE. IT ALSO HOSTS BI-ANNUAL MEETINGS OF ALL RESEARCHERS RECEIVING ITS FUNDING TO PROMOTE KNOWLEDGE EXCHANGE AND SYNERGY. IT USES THE SERVICES OF A MEDICAL RESEARCH CONSULTANT TO OVERSEE…