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  1. Article

    Open Access

    Agreement between results of meta-analyses from case reports and clinical studies, regarding efficacy and safety of idursulfase therapy in patients with mucopolysaccharidosis type II (MPS-II). A new tool for evidence-based medicine in rare diseases

    A preliminary exploratory study shows solid agreement between the results of case reports and clinical study meta-analyses in mucopolysaccharidosis Type I (MPS-I) adult patients. The aim of the present study i...

    Miguel Sampayo-Cordero, Bernat Miguel-Huguet… in Orphanet Journal of Rare Diseases (2019)

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