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

J.M. Heard, C. Vrinten, M. Schlander, C.M. Bellettato, C. van Lingen, M. Scarpa, G. Matthijs, M.C. Nassogne, F.G. Debray, D. Roland, T. Chamova, V. Kozich, J. Pavel, M. Zenker, C. Lampe, A.M. Das, J. Hennermann, S. Kolker, N. Weinhold, K. MohnikeS. Gruenert, A.M. Lund, M. Morales-Conejo, M. del Toro-Riera, L. Aldamiz-Echevarria, M.T. Garcia-Silva, M. Schiff, L. Gouya, P. Labrune, P. de Lonlay, N. Belmatoug, D.P. Germain, A. Cano, D. Dobbelaere, S. Jones, C. Dawson, P. Deegan, S. Santra, S. Vijay, D.P. Ramadza, I. Baric, T. Zigman, G. Pflieger, K. Szakszon, R. Kaposta, S. Gasperini, A. Burlina, G. Parenti, P. Strisciuglio, G. Ceccarini, MetabERN collaboration group, Maria Estela Rubio Gozalbo

Research output: Contribution to journalArticleAcademicpeer-review

Original languageEnglish
Article number3
Number of pages10
JournalOrphanet Journal of Rare Diseases
Volume15
Issue number1
DOIs
Publication statusPublished - 6 Jan 2020

Keywords

  • access to treatment
  • drugs
  • european reference network
  • hereditary metabolic diseases
  • inborn errors of metabolism
  • orphan medicinal product
  • Hereditary Metabolic Diseases
  • Access to treatment
  • Inborn errors of metabolism
  • Orphan medicinal product
  • DRUGS
  • European Reference Network

Cite this