CureCMT4J/Talia Duff Foundation

CureCMT4J is a non profit dedicated to expediting a treatment or cure for the ultra-rare disease, CMT4J. CMT4J is an inherited peripheral neuropathy that can cause profound muscle weakness in the limbs and respiratory muscles, leading to quadriplegia and early death due to respiratory failure. It can look a lot like ALS. CMT4J is a monogenic, autosomal recessive, ultra-rare disease involving the FIG4 gene. Currently, there is no available treatment; however, CureCMT4J created and funded successful pre-clinical Proof of Concept with AAV9/FIG4 Gene Therapy ((PMID: 33878035), in the Journal of Clinical Investigation. A CMT4J natural history study is on-going: CMT4J Natural History Study (NCT03810508). We are working to advance a first-in-human gene therapy trial. CureCMT4J is also spearheading and funding research in Drug Repurposing, ASO development, and continued research in revealing the translational complex pathways involved with the FIG4 gene. Research in rare diseases is translatable to other rare and more common diseases such as ALS, MS, and more common types of CMT. CureCMT4J also promotes awareness for the more than 10,000 rare diseases that exist--only 5% of them having an available treatment. Cure CMT4J / The 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 U.S. Internal Revenue Service.