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
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.
These meetings are a way to connect with VCP families around the world and share experiences. There is no agenda or drink required.
Because we have members all over the world, we will be offering two separate happy hours as follows:
Happy Hour 1: Wed, Apr 21, 2020 @ 8:00 PM Eastern Standard Time
Happy Hour 2: Sat, Apr 24, 2020 @ 3:00 PM Eastern Standard Time
We look forward to seeing you then!
To attend and get the zoom log-in info: Click one of the below sessions:
Confused about time zones? Click here for a time zone converter https://time.is/compare
VCP Scientific Conference
Virtual event: Register here