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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Vydáno v:Int J Environ Res Public Health
Hlavní autoři: Sampayo-Cordero, Miguel, Miguel-Huguet, Bernat, Malfettone, Andrea, Pérez-García, José Manuel, Llombart-Cussac, Antonio, Cortés, Javier, Pardo, Almudena, Pérez-López, Jordi
Médium: Artigo
Jazyk:Inglês
Vydáno: MDPI 2020
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Article
On-line přístup: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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