Jocelyn Duff is the Executive Director and co-founder of CureCMT4J, an all-volunteer non-profit dedicated to expediting a treatment or cure for Charcot Marie Tooth Disease Type 4J (CMT4J). Jocelyn and her husband, John, founded CureCMT4J in June, 2016 after receiving a diagnosis of CMT4J for their daughter, Talia, following a six-year diagnostic odyssey. CMT4J is a severe, progressive, inherited neuromuscular disease often compared to ALS.
At present there is no treatment or cure. In its initial 18 months of operation, CureCMT4J gathered world experts in CMT4J/FIG4 and gene therapy, raised over $1 million, and funded pre-clinical work at the Jackson Laboratory, establishing efficacy using gene therapy in CMT4J mouse models. A first-in-human gene therapy clinical trial is planned for 2020.
A physician assistant (PA) by training, Jocelyn has worked in Family Practice and Internal Medicine for over 20 years. She was a recipient of The Science Channel’s “Superheroes in Science” award, in June, 2017. Duff was also selected to speak at The Atlantic’ Live “Women in Science” symposium in Washington, D.C., discussing women-led scientific breakthroughs. Jocelyn mentors other rare disease families, connects scientists on relevant research projects, and holds advisory board positions at rare disease organizations. Her greatest joy is sharing adventures with her two daughters and husband.