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

29 Sept 2020

Abstract excerpt

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 with alpha-mannosidosis. It is a post-approval commitment to European marketing authorization for Velmanase alfa...

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PMID: 32993743
DOI: 10.1186/s13023-020-01549-8

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