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Characteristics of drugs for ultra-rare diseases versus drugs for other rare diseases in HTA submissions made to the CADTH CDR

BACKGROUND: It has been suggested that ultra-rare diseases should be recognized as distinct from more prevalent rare diseases, but how drugs developed to treat ultra-rare diseases (DURDs) might be distinguished from drugs for ‘other’ rare diseases (DORDs) is not clear. We compared the characteristic...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Richter, Trevor, Janoudi, Ghayath, Amegatse, William, Nester-Parr, Sandra
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2018
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5793441/
https://ncbi.nlm.nih.gov/pubmed/29386040
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-018-0762-1
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