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.
We need your help to identify all the doctors, practitioners and therapists who are currently treating VCP patients worldwide. You can help us build our VCP Medical Network by referring your doctor to us. This information will be used to create a Standard of Care, which will help in treating all VCP disease patients. This project is lead by Dr. Manish Korb and advised by our medical advisory board.
REFER YOUR DOCTOR OR THERAPIST
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, Jan 20, 2020 @ 8:00 PM Eastern Standard Time
Happy Hour 2: Sat, Jan 23, 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
The Treatment Story for
International Paget’s Awareness Day 2021
Paget's disease of the bone affects over half of VCP patients. The good news is that there are treatments available if it is detected early enough.
The Paget’s Association has made a series of short videos focusing on The Treatment Story, for Paget’s Awareness Day, 11th January 2021. Professor Stuart Ralston interviews Professors Graham Russell, Ian Reid and Mike Rogers, and Dr Roger Smith, to track the history and future of bisphosphonates. In addition, using animation, Dr Anna Daroszewska describes how bisphosphonates work, and patients, Linda and Keith, discuss their experiences. Learn more https://paget.org.uk/full-treatment-stories