The SPARKLE registry: protocol for an international prospective cohort study in patients with alpha-mannosidosis | Orphanet Journal of Rare Diseases | Full Text

The SPARKLE registry: protocol for an international prospective cohort study in patients with alpha-mannosidosis | Orphanet Journal of Rare Diseases | Full Text

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

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 dat...

Authors:Julia B. Hennermann, Nathalie Guffon, Federica Cattaneo, Ferdinando Ceravolo, Line Borgwardt, Allan M. Lund, Mercedes Gil-Campos, Anna Tylki-Szymanska and Nicole M. Muschol

Citation:Orphanet Journal of Rare Diseases 2020 15:271

Content type:Research

 

Published on: 29 September 2020

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