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Editorial
. 2022 May;45(3):381-382.
doi: 10.1002/jimd.12501. Epub 2022 Apr 7.

Lost in translation-Challenges in drug development for inherited metabolic diseases

Robin H Lachmann  1 , Marc C Patterson  2 , Sandra Sirrs  3
Affiliations
Editorial

Lost in translation-Challenges in drug development for inherited metabolic diseases

Robin H Lachmann et al. J Inherit Metab Dis. 2022 May.
No abstract available

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References

REFERENCES

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    1. Caetano R, Cordeiro Dias Villela Correa M, Villardi P, Almeida Rodrigues PH, Garcia Serpa Osorio-de-Castro C. Dynamics of patents, orphan drug designation, licensing, and revenues from drugs for rare diseases: the market expansion of eculizumab. PLoS One. 2021;16:e0247853. doi:10.1371/journal.pone.0247853
    1. Gamba S, Magazzini L, Pertile P. R&D and market size: who benefits from orphan drug legislation? J Health Econ. 2021;80:102522. doi:10.1016/j.jhealeco.2021.102522
    1. Abrahamyan L, Feldman BM, Tomlinson G, et al. Alternative designs for clinical trials in rare diseases. Am J Med Genet C Semin Med Genet. 2016;172:313-331. doi:10.1002/ajmg.c.31533
    1. Côté A, Keating B. What is wrong with orphan drug policies. Value Health. 2012;15:1185-1191. doi:10.1016/j.jval.2012.09.004

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