Cure VCP Disease, Inc., is a patient advocacy organization dedicated to driving the development of a cure for valosin-containing protein (VCP) associated multisystem proteinopathy (MSP), also known as IBMPFD (Inclusion Body Myopathy, Early Onset Paget’s Disease of Bone and Frontotemporal Dementia). The disease is an adult-onset, hereditary, autosomal dominant disease caused by a pathogenic variant of the VCP gene on chromosome 9. It can affect any combination of a patients’ muscles, bones and brain and can cause degenerative diseases including ALS, frontotemporal dementia, Parkinson’s, and Charcot-Marie Tooth.

In 2018, we identified the need to identify and organize patients and families affected by mutations in the VCP gene (p97). We have moved quickly in that time by creating the first VCP disease-exclusive patient registry, facilitating a Patient Listening Session with the FDA and conducting the first VCP-focused Scientific Conference in North America.