May 5, 2023
We are thrilled to announce a publication in Annals of Clinical and Translational Neurology that offers provisional practice recommendations for the management of myopathy in VCP-associated multisystem proteinopathy.
Led by Dr. Bhaskar Roy of Yale and assisted by Allison Peck of Cure VCP Disease, a collaborative group of 18 top physicians worked together to compile these recommendations.
The team included experts from 17 different institutions and 6 countries, making this a truly global effort to improve medical care for VCP patients with muscle weakness.
The aim of this working group was to develop a best practice set of provisional recommendations for VCP myopathy that can be easily implemented across the globe.
Valosin-containing protein (VCP)-associated multisystem proteinopathy (MSP) is a rare genetic disorder that affects the autophagy pathway leading to various combinations of myopathy, bone diseases, and neurodegeneration.
Ninety percent of VCP patients suffer from muscle weakness known as myopathy. Therefore, this guideline fills a critical need among patients and their providers to improve the diagnosis and ongoing medical care for VCP patients with myopathy.
The publication provides a detailed review of diagnosing this condition, follow-up testing, and treatment recommendations.
The working group concluded that standardized management of VCP myopathy will optimize patient care and help future research initiatives.
Click here to read the publication.
Cure VCP Disease expresses our sincere gratitude to all the expert physicians who volunteered their time to this work:
Bhaskar Roy, Teresinha Evangelista, Gerald Pfeffer, Leo Wang, Jordi Diaz-Manera, Manisha Korb, Matthew P. Wicklund, Margherita Milone, Miriam Freimer, Hani Kushlaf, Rocio-Nur Villar-Quiles, Tanya Stojkovic, Merrilee Needham, Johanna Palmio, Thomas E. Lloyd, Benison Keung, Tahseen Mozaffar, Conrad Chris Weihl, Virginia Kimonis
We are a patient advocacy organization driving efforts to discover a cure for neurodegenerative diseases associated with VCP including IBMPFD, dementia, Parkinson's, CMT, 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.