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Molecular Genetics and Metabolism|January 17, 2018
Agreement between the results of meta-analyses from case reports and from clinical studies regarding the efficacy of laronidase therapy in patients with mucopolysaccharidosis type I who initiated enzyme replacement therapy in adult age: An example of case reports meta-analyses as an useful tool for evidence-based medicine in rare diseasesMiguel Sampayo-Cordero, Bernat Miguel-Huguet, Almudena Pardo-Mateos, et al.
Internal Medicine Journal|February 4, 2026
Canakinumab as effective therapy in anakinra-refractory Schnitzler syndrome: a case-based reviewSergio De Santos Belinchón, Cristina Ausín García, Pablo Daniel Lorenzo Barcia, et al.
Orphanet Journal of Rare Diseases|October 23, 2019
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 diseasesMiguel Sampayo-Cordero, Bernat Miguel-Huguet, Almudena Pardo-Mateos, et al.
Biomedicines|March 28, 2024
Women with Gaucher DiseaseMaria Del Mar Meijon-Ortigueira, Isabel Solares, Cecilia Muñoz-Delgado, et al.
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