CureCMT4J/Talia Duff Foundation is a 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 for which there is currently no available treatment or cure.
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. “Proof of concept” was achieved in CMT4J mouse models using AAV9/FIG4 gene therapy, showing efficacy and safety. CureCMT4J is now working with a biopharma company, Neurogene, toward a gene therapy clinical trial and a natural history study–a clinical study intended to better understand the disease. For more information visit: https://www.neurogene.com/patients-and-families/#CMT4J
CureCMT4J continues to research other pathways toward treatments for CMT4J, including drug repurposing, small molecule development, ASO therapies and others. See our “Research” page for more information.
Who We Are
Jocelyn Duff, Co-Founder, Executive Director – Jocelyn is Mom to her daughter, Talia, who has CMT4J. Jocelyn and her husband, John, founded CureCMT4J in June, 2016 after finally receiving a diagnosis of CMT4J for Talia, following a six-year diagnostic odyssey. Since 2016 she has led the efforts of CureCMT4J in furthering treatments for CMT4J. Jocelyn was a physician assistant in internal medicine and family practice for 20 years before starting CureCMT4J. To read more about how Jocelyn is fighting for and spreading awareness of CMT4J, as well as other rare diseases, visit https://rareparent.com/
CureCMT4J has both a Scientific Advisory Board, as well as a general Board of Directors. 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.