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...

Descripció completa

Guardat en:
Dades bibliogràfiques
Publicat a:Orphanet J Rare Dis
Autors principals: Hennermann, Julia B., Guffon, Nathalie, Cattaneo, Federica, Ceravolo, Ferdinando, Borgwardt, Line, Lund, Allan M., Gil-Campos, Mercedes, Tylki-Szymanska, Anna, Muschol, Nicole M.
Format: Artigo
Idioma:Inglês
Publicat: BioMed Central 2020
Matèries:
Research
Accés en línia: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
Etiquetes: Afegir etiqueta
Sense etiquetes, Sigues el primer a etiquetar aquest registre!

Ítems similars