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

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

Abstract 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...

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Bibliographic Details
Main Authors: Jean-Michel Heard, Charlotte Vrinten, Michael Schlander, Cinzia Maria Bellettato, Corine van Lingen, Maurizio Scarpa, the MetabERN collaboration group
Format: Article
Language:English
Published: BMC 2020-01-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Orphan medicinal product
Access to treatment.
European Reference Network.
Hereditary Metabolic Diseases.
Inborn errors of metabolism.
Online Access:https://doi.org/10.1186/s13023-019-1280-5

Internet

https://doi.org/10.1186/s13023-019-1280-5

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