About CureCMT4J

ALL-VOLUNTEER NON PROFIT RESEARCH ORGANIZATION

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 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. 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 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 working 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.