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Essential list of medicinal products for rare diseases: recommendations from the IRDiRC Rare Disease Treatment Access Working Group.

William A GahlDurhane Wong-RiegerVirginie HivertRachel YangGalliano ZanelloStephen Groft
Published in: Orphanet journal of rare diseases (2021)
The proposed list of essential medicinal products for rare diseases is intended to initiate discussion and collaboration among patient advocacy groups, health care providers, industry and government agencies to enhance access to appropriate medicines for all rare disease patients throughout the world.
Keyphrases
  • healthcare
  • end stage renal disease
  • ejection fraction
  • newly diagnosed
  • chronic kidney disease
  • peritoneal dialysis
  • case report
  • combination therapy
  • affordable care act