About CureCMT4J

ALL-VOLUNTEER RARE DISEASE RESEARCH NONPROFIT

CureCMT4J/Talia Duff Foundation is an all-volunteer non-profit organization dedicated to developing a treatment or cure for CMT4J (Charcot Marie Tooth disease type 4J [CMT is also known as Hereditary and Sensory Motor Neuropathy]). CMT4J is a deadly, devastating ultra-rare disease similar to ALS.

CURECMT4J HAS DRIVEN THE RESEARCH AND FUNDING TOWARD A FIRST-IN-HUMAN AAV9 GENE THERAPY!
CLINICAL TRIALS ARE EXPECTED TO BEGIN IN LATE SPRING/SUMMER OF 2026.
VISIT THE NIH CLINICAL TRIALS WEBSITE HERE FOR MORE INFORMATION, OR REACH OUT TO jocelyn@curecmt4j.org

Founded in June, 2016, CureCMT4J’s mission is to generate awareness and support for the expedited therapeutic development of a treatment or cure for CMT4J.

CureCMT4J views its secondary mission as an advocacy organization, providing a voice and platform for rare disease advocacy and research through speaking engagements, rare disease conferences and social media education, awareness and fundraising campaigns.

Since 2016 CureCMT4J has spearheaded and furthered preclinical research and IND-enabling efforts for a first-in-human AAV9 gene therapy trial for FIG4/CMT4J. Preclinical “Proof of concept” was achieved in CMT4J mouse models using AAV9/FIG4 gene therapy, showing efficacy and safety. This research was published in the Journal of Clinical Investigation.

CureCMT4J continues to research other pathways toward treatments for CMT4J, including drug repurposing, novel affiliated gene pathways, small molecule development, ASO therapies and others. See our “Research” page for more information.

WHO WE ARE
Jocelyn and John Duff, Co-Founders/Parents of a child with CMT4J

Jocelyn is Mom to her daughter, Talia, who has CMT4J. Jocelyn serves as Executive Director for CureCMT4J, which was founded in June, 2016 after Talia’s diagnosis of CMT4J, following a six-year misdiagnosis of CIDP. Since 2016 she has led the efforts of CureCMT4J in furthering treatments for CMT4J. Jocelyn was a physician assistant (PA) in internal medicine and family practice for 20 years before leaving her career to run the foundation and care for her daughter.

CureCMT4J has a team of scientific investigators who are experts in CMT, the FIG4 Gene implicated in CMT4J, and in translational medicine for rare diseases. The CureCMT4J Board is an all-volunteer, working Board that leads our nonprofit with expertise and urgency. Our Board members possess unique skillsets in small business and startup management, FDA expertise, research and development, fundraising, communications and medicine.

CureCMT4J/Talia Duff Foundation Inc. (Tax ID: 81-3019217) is a Massachusetts corporation. Federal tax exempt status as a public charity under Section 501(c)(3) has been approved by the US Internal Revenue Service.