Stepien, Karolina-M; Thomas, Sophie; Hennermann, Julia-B; Lampe, Christina; Muschol, Nicole-M; Ballesta-Martínez, María-Juliana; Cruz, Jordi; López-Rodríguez, Mónica; Barth, Anneliese; Magner, Martin; Lund, Allan-M; Plaiasu, Vasilica; Ballabeni, Andrea; Dona, Francesca; Morgan, Heather-M; Guffon, Nathalie
(BMC, 2025-05-07)
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 ...