We are a patient advocacy organization driving efforts to find a cure for diseases caused by a mutation of the VCP gene including IBMPFD 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

Giving Campaign

Research

Find out how you can get involved in participating in the latest research to help advance progress towards treatments for VCP disease.

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Patient Registry

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Resources

Find out more about patient and caregiver support groups, educational information and other documents.

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Give Now

Support our efforts at Cure VCP Disease, Inc. by giving now. Any donation amount is much appreciated

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Patient-Powered Research

Natural History Study

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Cure VCP Disease and Nationwide Children's Hospital are partnering to lear how VCP Disease change's peoples stength and movements and affects their daily life. This info will help improve care and plan future research studies.

To sign-up contact Lindsay Alfano at

What is VCP Disease?

VCP Disease is a rare genetic disease, which can affect a patient's muscles, bones and brain. It is an autosomal dominant neuromuscular disease. The exact number of patients currently diagnosed with this disease is unknown worldwide. Leading researchers believe there is a significant number of patients with the VCP mutation being misdiagnosed as Limb-girdle muscular dystrophy, myositis or other myopathies.

VCP Disease is caused by a mutation in a gene called Valosin Containing Protein (VCP or p97). The VCP gene provides instructions for producing the VCP enzyme. The VCP enzyme is involved in a wide variety of important cellular activities such as cell division and mitochondrial function. One of VCP's most critical jobs is to help break down proteins that are abnormal or no longer needed. When the VCP gene is mutated, these cellular functions are disrupted leading to several possible symptoms and disorders.

VCP Disease primarily manifests as IBMPFD, which identifies the three primary disorders originally associated with the disease: Inclusion Body Myopathy, Paget's Disease of Bone, and Frontotemporal Dementia. Researchers also categorize this disease as Multisystem Proteinopathy (MSP1) because it is a protein disorder that affects multiple body systems. A person with VCP Disease may have one, two, or all of these disorders. 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.

A genetic connection has now been established between VCP Disease and more common disorders such as Amyotrophic lateral sclerosis (ALS), Parkinsonism Disease, hereditary spastic paraplegia, Charcot-Marie-Tooth disease, cardiomyopathy, cataracts, and neuropathy. Learning more about VCP Disease may not only help advance the treatment of IBMPFD but also several disorders.