Enable Our 2023 Scientific Research Agenda
We're accelerating the scientific progress of VCP disease
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Cure VCP Disease has an ambitious scientific research agenda to drive clinical trial readiness for future therapeutics. Before a cure can be found, a toolbox must be in place that can assist scientists as well as the FDA in confirming that potential treatments and therapeutics will have efficacy.
Our goal is to create a toolbox, owned by Cure VCP Disease, that can be utilized by the global community interested in VCP disease. Our tools won't be tied down at a particular company or academic institution. These tools will be available to ALL in order to optimize the speed of development and knowledge.
It is time to create research models that can quickly and efficiently aid in the drug discovery process. If we want treatments for VCP disease, we need to invest in drug discovery. Our research agenda is broken into 4 initiatives:
1) Preclinical Toolbox - Create tools to advance scientific discovery, including patient-derived cellular and animal models owned by Cure VCP Disease.
2) Longitudinal Clinical Studies - Fund Year 3 of our Functional Measures Study at Nationwide Children's Hospital and continue development of video measures for future FDA and clinical trial use.
3) Treatment Discovery - Begin funding a 3-year gene editing program.
4) Awareness & Education - Continue to host patient and scientific conferences and support on-going patient advocacy efforts.
Please share our downloadable one-page flyer on our research agenda
We welcome the chance to meet with you, your friends, family, and colleagues who might want to contribute. If interested, please feel free to schedule time on my calendar to discuss further: https://bit.ly/talkvcp. There is nothing more important to me than finding a cure for VCP disease, and we need your engagement to make this happen.
About Cure VCP Disease:
Cure VCP Disease, Inc., is a patient advocacy organization dedicated to driving the development of a cure for valosin-containing protein (VCP) associated multisystem proteinopathy (MSP), also known as IBMPFD (Inclusion Body Myopathy, Early Onset Paget’s Disease of Bone and Frontotemporal Dementia). The disease is an adult-onset, hereditary, autosomal dominant disease caused by a pathogenic variant of the VCP gene on chromosome 9. It can affect any combination of a patient’s muscles, bones, and brain and can cause degenerative diseases including ALS, frontotemporal dementia, Parkinson’s, and Charcot-Marie Tooth.
100% of donations are used to enable Cure VCP Disease, Inc. to fund research, education, and awareness initiatives. Cure VCP Disease, Inc. has the right to redirect charitable contributions if the program goal has been met and/or is overfunded. The funds will be applied to the organization's greatest needs in research and program development.