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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Bibliographic Details
Main Authors: Miguel Sampayo-Cordero, Bernat Miguel-Huguet, Andrea Malfettone, José Manuel Pérez-García, Antonio Llombart-Cussac, Javier Cortés, Almudena Pardo, Jordi Pérez-López
Format: Article
Published: MDPI AG 2020-09-01
Series:International Journal of Environmental Research and Public Health
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