The SPARKLE registry: protocol for an international prospective cohort study in patients with alpha-mannosidosis

BACKGROUND: Alpha-mannosidosis is a lysosomal storage disorder caused by reduced enzymatic activity of alpha-mannosidase. SPARKLE is an alpha-mannosidosis registry intended to obtain long-term safety and effectiveness data on the use of velmanase alfa during routine clinical care in patients with al...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Hennermann, Julia B., Guffon, Nathalie, Cattaneo, Federica, Ceravolo, Ferdinando, Borgwardt, Line, Lund, Allan M., Gil-Campos, Mercedes, Tylki-Szymanska, Anna, Muschol, Nicole M.
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2020
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7525940/
https://ncbi.nlm.nih.gov/pubmed/32993743
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-020-01549-8
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