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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

BACKGROUND: The European Medicine Agency granted marketing approval to 164 orphan medicinal products for rare diseases, among which 28 products intended for the treatment of hereditary metabolic diseases. Taking advantage of its privileged connection with 69 healthcare centres of excellence in this...

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Détails bibliographiques
Publié dans:Orphanet J Rare Dis
Auteurs principaux: Heard, Jean-Michel, Vrinten, Charlotte, Schlander, Michael, Bellettato, Cinzia Maria, van Lingen, Corine, Scarpa, Maurizio
Format: Artigo
Langue:Inglês
Publié: BioMed Central 2020
Sujets:
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC6945588/
https://ncbi.nlm.nih.gov/pubmed/31907071
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-019-1280-5
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