Rare diseases affect one in 10 people.

CMT4J is a rare, progressive, neurological disease similar to ALS.

CureCMT4J is a non-profit created by parents of Talia, a 14 year-old girl with severe, early-onset CMT4J. CureCMT4J is the only entity working toward a treatment or cure for this deadly disease. YOU can help us to get there. 

$1,379,148
OF OUR $2,200,000 GOAL

CURECMT4J IS DRIVING RESEARCH TOWARD TREATMENT OR CURE

Our researchers working together from The Jackson Laboratory, UT Southwestern, and University of Michigan have shown great success in using gene therapy to treat CMT4J mouse models! We’re working hard on getting it into a clinical trial! 

Gene Therapy

Drug Repurposing

Small Molecule Treatment

To learn more about our research visit our Research Pathway page

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