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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
BACKGROUND: 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 is to confirm previous results in another patient population, suffering from muc...
Autores principales: | Sampayo-Cordero, Miguel, Miguel-Huguet, Bernat, Pardo-Mateos, Almudena, Malfettone, Andrea, Pérez-García, José, Llombart-Cussac, Antonio, Cortés, Javier, Moltó-Abad, Marc, Muñoz-Delgado, Cecilia, Pérez-Quintana, Marta, Pérez-López, Jordi |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6805333/ https://www.ncbi.nlm.nih.gov/pubmed/31639024 http://dx.doi.org/10.1186/s13023-019-1202-6 |
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