Treatment Information & Other Resources

Currently there is no treatment or cure for CMT4J. However, The Talia Duff Foundation is on a path to test and use gene therapy to treat and cure CMT4J.

Many clinicians and scientists believe that Gene Therapy holds promise for a cure for CMT4J and many other rare diseases. Gene therapy has already shown success in other diseases and is currently being used in over 600 clinical trials throughout the world. We are truly on the cusp of enormous breakthroughs using gene therapy to treat and cure many diseases. Rare and ultra-rare diseases represent ideal diseases to treat with gene therapy, as most are monogenic (involving only one gene).

NIH: Genes/Gene Therapy explained
https://newsinhealth.nih.gov/issue/dec2013/feature1

NIH: 2014 update for abolishing special review for gene therapy trials
http://www.sciencemag.org/news/2014/05/nih-will-no-longer-require-special-review-us-gene-therapy-trials

Parent-driven models of driving the science/funding for rare diseases:
http://www.hannahshopefund.org/
http://www.curebatten.org/
http://jonahsjustbegun.org/

Relevant paper on parent-driven research models:
http://csmres.co.uk/cs.public.upd/article-downloads/Ultra-rare-diseases.pdf

Drug development roadmap, from Global Genes:
https://globalgenes.org/wp-content/uploads/2016/02/Toolkit-Roadmap_web_112415.pdf

Neurotoxic Medications to Avoid:
http://www.hnf-cure.org/wp-content/uploads/2015/06/neurotoxins.pdf
http://www.cmtausa.org/resource-center/treatment-management/neurotoxic-medications/

Overview of CMT (not the “4J” type):
http://www.hnf-cure.org/about-us/cmt/