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Availability, accessibility and delivery to patients of the 28 orphan medicines approved by the European Medicine Agency for hereditary metabolic diseases in the MetabERN network.
Heard, Jean-Michel; Vrinten, Charlotte; Schlander, Michael; Bellettato, Cinzia Maria; van Lingen, Corine; Scarpa, Maurizio.
Affiliation
  • Heard JM; MetabERN, Regional Coordinating Center for Rare Diseases, Udine University Hospital, Piazzale Santa Maria della Misericordia, 15, 33100, Udine, Italy. jean-michel.heard@metab.ern-net.eu.
  • Vrinten C; Imperial College Business School, London, UK.
  • Schlander M; Division of Health Economics, German Cancer Research Center, Heidelberg, Germany.
  • Bellettato CM; MetabERN, Regional Coordinating Center for Rare Diseases, Udine University Hospital, Piazzale Santa Maria della Misericordia, 15, 33100, Udine, Italy.
  • van Lingen C; MetabERN, Regional Coordinating Center for Rare Diseases, Udine University Hospital, Piazzale Santa Maria della Misericordia, 15, 33100, Udine, Italy.
  • Scarpa M; MetabERN, Regional Coordinating Center for Rare Diseases, Udine University Hospital, Piazzale Santa Maria della Misericordia, 15, 33100, Udine, Italy.
Orphanet J Rare Dis ; 15(1): 3, 2020 01 06.
Article in En | MEDLINE | ID: mdl-31907071
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Orphan Drug Production / Metabolic Diseases / Metabolism, Inborn Errors Limits: Humans Language: En Journal: Orphanet J Rare Dis Journal subject: MEDICINA Year: 2020 Type: Article Affiliation country: Italy
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Orphan Drug Production / Metabolic Diseases / Metabolism, Inborn Errors Limits: Humans Language: En Journal: Orphanet J Rare Dis Journal subject: MEDICINA Year: 2020 Type: Article Affiliation country: Italy