Neurogene, a leading company developing genetic medicines for people with rare, devastating neurological diseases, announced today that it is recruiting patients for a natural history study for CMT4J.
“Key data now being collected to support endpoint assessment for future gene therapy clinical trial”
CMT4J is a rare recessive type of CMT, which can be quite similar to ALS – so rare that there are only roughly 20 known cases of patients with CMTJ4. Neurogene has partnered with UT Southwestern to explore potential genetic therapies for CMT4J, and is currently looking to gain a deep understanding of the natural history of the disease. This study aims to provide just that.
Natural history studies help researchers understand the course of a disease’s progression so that clinical trials can be more readily executed and their efficacy measured. Without natural history studies, it can be very difficult to determine if any treatment is changing the rate of degenerative progression. Gathering natural history studies for each type of CMT is vitally important to help benchmark potential therapies.
Read the press release, more about the natural history study for CMT4J, CMT4J and Neurogene. You can also learn more about natural history studies for your type of CMT.