We are a patient advocacy organization driving efforts to discover a cure for neurodegenerative diseases associated with VCP including IBMPFD, dementia, and ALS. We encourage patients and doctors to connect with us. We are committed to collaborating on research and advocacy initiatives and helping patients and their families.
Natural History Study
Cure VCP Disease and Nationwide Children's Hospital are partnering to learn how VCP disease change's peoples strength and movements and affects their daily life. This information will help improve care and plan future research studies. Now enrolling patients internationally and remote-only.
To sign-up contact Lindsay Alfano at NMDtrialinfo@nationwidechildrens.org
What is VCP Disease?
VCP associated multi-system proteinopathy
VCP disease is a rare genetic disease, which can affect a patient's muscles, bones and brain. It is an autosomal dominant, neuromuscular disease. This means that there is a 50% chance a VCP patient can pass the mutated gene on to a child. Only a genetic test can confirm VCP disease.
VCP disease is caused by a mutation in a gene called Valosin Containing Protein (VCP or p97). VCP disease is medically known as multisystem proteinopathy (MSP1) because it is a protein disorder that affects multiple body systems. A person with a VCP disease may develop one or several of the following disorders: inclusion body myopathy, Paget's disease of bone, frontotemporal dementia, amyotrophic lateral sclerosis (ALS), Parkinsonism, hereditary spastic paraplegia, Charcot-Marie-Tooth disease, cardiomyopathy, cataracts, and neuropathy.
There are no known cures or treatments for myopathy or dementia, but there is an approved treatment for Paget's disease of bone and there are therapies that may help delay the symptoms. It is important for a person with a VCP disease to regularly see a doctor for check-ups. See our published care guidelines here: https://ojrd.biomedcentral.com/articles/10.1186/s13023-022-02172-5
The exact number of patients currently diagnosed with this disease is unknown worldwide, but as more patients are identified and connect with us, the more we learn. The more we learn, the closer we get to treatments. Learning more about VCP disease may not only help advance the treatment of VCP disease but also other diseases.
Free Resource on Care Guidelines
Tired of explaining your rare disease to health care providers? Help is on the way!
Request these free business cards to give to your health care providers to help them care for you. Since many doctors and nurses may only see one or two VCP patients in their lifetime, getting proper care is hard and complex when you have VCP disease. Fortunately, a team of international doctors collaborated on establishing clinical care guidelines to help doctors quickly understand the symptoms, testing, and therapies to consider for you.
Patient Support and Events
These meetings are a way to connect with VCP families around the world and share experiences. There is no agenda and no drink is required.
Wednesday, August 10, 2022 08:00 PM Eastern Time (US and Canada)
Wednesday, August 24, 2022 08:00 PM Eastern Time (US and Canada)
We look forward to seeing you then!
To attend and get the log-in info: Click below
Confused about time zones? Click here for a time zone converter https://time.is/compare