Utilization of CoRDS registry to monitor quality of life in patients with VCP multisystem proteinopathy

Abstract Background VCP disease, also known as multisystem proteinopathy, is a rare, autosomal dominant, adult-onset, neuromuscular disease that is caused by variants in the valosin-containing protein (VCP) gene. VCP disease may exhibit one or more of the following primary features: inclusion body m...

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Autori principali:	Eiman Abdoalsadig, Merwa Hamid, Allison Peck, Leepakshi Johar, Virginia Kimonis
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	BMC 2025-04-01
Serie:	Orphanet Journal of Rare Diseases
Soggetti:	VCP Inclusion body myopathy Paget’s disease of bone Frontotemporal dementia (IBMPFD) Amyotrophic lateral sclerosis (ALS) Multisystem proteinopathy
Accesso online:	https://doi.org/10.1186/s13023-025-03567-w
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