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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 diseases.
Sampayo-Cordero M, Miguel-Huguet B, Pardo-Mateos A, Moltó-Abad M, Muñoz-Delgado C, Pérez-López J. Sampayo-Cordero M, et al. Among authors: pardo mateos a. Mol Genet Metab. 2018 Feb;123(2):69-75. doi: 10.1016/j.ymgme.2018.01.002. Epub 2018 Jan 5. Mol Genet Metab. 2018. PMID: 29336994 Review.
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.
Sampayo-Cordero M, Miguel-Huguet B, Pardo-Mateos A, Malfettone A, Pérez-García J, Llombart-Cussac A, Cortés J, Moltó-Abad M, Muñoz-Delgado C, Pérez-Quintana M, Pérez-López J. Sampayo-Cordero M, et al. Among authors: pardo mateos a. Orphanet J Rare Dis. 2019 Oct 21;14(1):230. doi: 10.1186/s13023-019-1202-6. Orphanet J Rare Dis. 2019. PMID: 31639024 Free PMC article.