CMT4J is an ultra-rare, progressive
neurological disease similar to ALS.
WE HAVE IND APPROVAL FROM THE FDA FOR OUR
AAV9/FIG4 GENE THERAPY CLINICAL TRIAL!
A CLINICAL TRIAL IS EXPECTED TO BEGIN Q2, 2026
IF YOU OR A LOVED ONE HAS CMT4J, CONTACT US at jocelyn@curecmt4j.org FOR MORE INFO
CureCMT4J is an all-volunteer non-profit created by parents of Talia, who has severe, early-onset CMT4J.
CureCMT4J’S MISSION IS TO DEVELOP LIFE-SAVING, LIFE-CHANGING TREATMENTS FOR CMT4J.
YOU can help us to get there!
CURECMT4J IS DRIVING RESEARCH TOWARD TREATMENTS!
OUR CMT4J GENE THERAPY CLINICAL TRIAL IS READY TO BEGIN in late SPRING/SUMMER, 2026
OUR CMT4J NATURAL HISTORY STUDY IS FULLY ENROLLED!
IF YOU OR A LOVED ONE HAS CMT4J CONTACT JOCELYN@CURECMT4J.ORG FOR MORE INFO
Gene Therapy
Drug Repurposing
To learn more about our research visit our Research Pathway page
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