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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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Détails bibliographiques
Publié dans:Int J Environ Res Public Health
Auteurs principaux: Sampayo-Cordero, Miguel, Miguel-Huguet, Bernat, Malfettone, Andrea, Pérez-García, José Manuel, Llombart-Cussac, Antonio, Cortés, Javier, Pardo, Almudena, Pérez-López, Jordi
Format: Artigo
Langue:Inglês
Publié: MDPI 2020
Sujets:
Accès en ligne: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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