NAME OF ORGANIZATION: Cure VCP Disease, Inc.
WHAT LGMD SUB-TYPE DOES YOUR ORGANIZATION FOCUSES ON:
VCP diseases and specifically Inclusion Body Myopathy associated with Paget’s disease of bone and Frontotemporal Dementia (IBMPFD).
IF YOU FOCUS ON A SUB-TYPE, IS THERE A REGISTRY AND HOW CAN PATIENTS JOIN?
Is your organization a Non-Profit? If so, what type:
Yes, we are a 501(c)(3) public non-profit organization
WHAT INSPIRED THE CREATION OF YOUR ORGANIZATION:
There was no formal organization in existence to organize and provide awareness of VCP diseases to patients and researchers. I am a patient myself and had lost my mom to the disease and felt that I could not stand idly by to wait and hope for things to happen in the research community. As a patient population, we needed to be active participants to drive positive outcomes, but most importantly to identify the patient population.
WHAT IS YOUR ORGANIZATION’S MISSION:
Cure VCP Disease, Inc. was formed to drive efforts to cure diseases related to mutations of the Valosin Containing Protein gene. This includes the disease Inclusion Body Myopathy associated with Paget’s disease of bone and Frontotemporal Dementia (IBMPFD). We aim to provide global education and awareness of VCP diseases to doctors, researchers, investors, patients, caregivers and the general public.
WHAT SERVICES DOES YOUR ORGANIZATION PROVIDE:
We maintain a global patient registry of VCP disease patients. We provide hope and education to our patient population through conferences, e-mail newsletters, online resources, and webinars.
WHAT IS YOUR ORGANIZATION MOST PROUD OF:
We started up in January 2018 and by April 2019 conducted the inaugural VCP disease patient and caregiver conference. We had about 60 patients, caregivers and researchers in attendance. All of the recorded sessions are available on our Cure VCP Disease YouTube channel.
WHAT DO YOU WANT THE WORLD TO KNOW ABOUT YOUR ORGANIZATION:
Many of our patients are misdiagnosed as LGMD or Inclusion Body Myositis. The journey for a correct diagnosis can be stressful for many patients with neuromuscular diseases. Our goal is to partner with all adult-onset neuromuscular organizations to drive awareness, education and best practices. We all face many of the same challenges due to our diseases and though the science of our diseases may be different, we all have to be in this together.
HOW CAN PEOPLE BECOME INVOLVED IN SUPPORTING YOUR ORGANIZATION:
We are trying to create an Ambassador program to reach out to patients, caregivers and the scientific community. We do not know the prevalence of VCP diseases and identifying more patients is critical to understanding the global impact.
WHAT IS THE BEST WAY TO CONTACT YOUR ORGANIZATION:
Website, Facebook group or E-mail
IS THERE ANYTHING ELSE YOU’D LIKE TO ADD?
The key question to differentiating between VCP disease and maybe a LGMD mutation is the hereditary question, “Do other family members exhibit muscle weakness, bone disease and/or dementia?” VCP disease is autosomal dominant and most times passed through families and might present with different symptoms. My mother, aunt and two uncles (4 out of 6 siblings) had/have VCP disease. Since I am a carrier of the mutation, it is 50/50 that my offspring could be affected. The VCP gene is now included on most neuromuscular disease genetic testing panels.