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

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

Abstract 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...

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Bibliographic Details
Main Authors: Julia B. Hennermann, Nathalie Guffon, Federica Cattaneo, Ferdinando Ceravolo, Line Borgwardt, Allan M. Lund, Mercedes Gil-Campos, Anna Tylki-Szymanska, Nicole M. Muschol
Format: Article
Language:English
Published: BMC 2020-09-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Alpha-mannosidosis
Recombinant alpha-mannosidase
Velmanase alfa
Patient registry
Enzyme-replacement therapy
Online Access:http://link.springer.com/article/10.1186/s13023-020-01549-8

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http://link.springer.com/article/10.1186/s13023-020-01549-8

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