Neurofibromatosis type 1, also known as NF1, is a genetic disease caused by a mutation in the NF1 gene. It can lead to skin discoloration and the growth of tumors (called neurofibromas) on nerves anywhere in the body. These tumors are typically non-cancerous (benign), but if they become too large, they can cause other issues.
A plexiform neurofibroma, or PN, is a specific type of tumor that grows extensively along a nerve. As the PN grows, it can put pressure on the nerves, causing pain and loss of function in certain areas.
The ReNeu study is researching the effectiveness of the study drug at improving a PN that is causing significant issues in people with NF1.
Watch the video below to hear Dr. Mary Smith and SpringWorks Therapeutics present an overview of NF1 and the ReNeu trial, in collaboration with the Neurofibromatosis Network.