The Value of Case Reports in Systematic Reviews from Rare Diseases. The Example of Enzyme Replacement Therapy (ERT) in Patients with Mucopolysaccharidosis Type II (MPS-II)

Background: Case reports are usually excluded from systematic reviews. Patients with rare diseases are more dependent on novel individualized strategies than patients with common diseases. We reviewed and summarized the novelties reported by case reports in mucopolysaccharidosis type II (MPS-II) pat...

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Detalhes bibliográficos
Publicado no:Int J Environ Res Public Health
Main Authors: Sampayo-Cordero, Miguel, Miguel-Huguet, Bernat, Malfettone, Andrea, Pérez-García, José Manuel, Llombart-Cussac, Antonio, Cortés, Javier, Pardo, Almudena, Pérez-López, Jordi
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7558586/
https://ncbi.nlm.nih.gov/pubmed/32927819
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijerph17186590
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