Evolution of mobility, pain/discomfort, self-care, and mental health in patients with alpha-mannosidosis: an international caregiver and patient survey

Abstract Background Alpha-mannosidosis is a rare recessive lysosomal storage disorder with progressive multi-systemic impacts. In the absence of standardized monitoring protocols, there is insufficient understanding of disease progression over time. This study explored the evolution of the burden of...

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Autori principali:	Karolina M. Stepien, Sophie Thomas, Julia B. Hennermann, Christina Lampe, Nicole M. Muschol, Maria Juliana Ballesta-Martínez, Jordi Cruz, Mónica López-Rodríguez, Anneliese Barth, Martin Magner, Allan M. Lund, Vasilica Plaiasu, Andrea Ballabeni, Francesca Donà, Heather M. Morgan, Nathalie Guffon
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	BMC 2025-05-01
Serie:	Orphanet Journal of Rare Diseases
Soggetti:	Alpha-mannosidosis Survey Walking Pain Self-care Mental health
Accesso online:	https://doi.org/10.1186/s13023-025-03694-4
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