Driving efforts to find a cure for diseases caused by a mutation of the VCP gene including IBMPFD and ALS.

WHAT IS VCP DISEASE?

VCP Disease is a rare genetic disease, which can affect a patient's muscles, bones and brain.  There are less than 500 people currently diagnosed with this disease worldwide.  Leading researchers believe there may be significantly more patients with this mutation, but they are being misdiagnosed as MS, MD, ALS, myositis or myopathy.

 

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.  A person with IBMPFD 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.  Early testing is very important.

A genetic connection has now been established between IBMPFD and more common disorders such as ALS and Parkinson's Disease.  Learning more about IBMPFD may not only help advance the treatment of IBMPFD but also several disorders.

WHO ARE WE?

Our patient advocacy group was founded to drive efforts in finding a cure for VCP Disease.  Our organization has four primary initiatives:

1.  Educate:  Promote  awareness of VCP Disease in the medical community and among patients.

2.  Identify:  Manage a registry of patients with VCP disease.  Having an accurate database is the only way to advance research, funding and understanding of the disease.  We encourage all patients to register.

3.  Support:  Provide support and resources for patients, caregivers and their doctors.  We are also working with clinicians and researchers to advance possible treatments for VCP Disease. 

4.  Give:  If you are interested in getting involved, please contact us.  There are many ways to volunteer and give.

There are some very promising advancements in understanding this disease, and new potential treatments are on the horizon.  If you think that you may have this disease or you would like to help, please reach out to us at curevcpdisease@gmail.com.

2019 PATIENT CONFERENCE

The Cure VCP Disease Patient & Caregiver Conference takes place April 12-13, 2019 at Washington University in St. Louis, Missouri. 

We will have a live stream of the event and please review the agenda and times on our Patient Conference page.

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EIN NO.: 82-4368871