Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Editorial
. 2022 Sep 28:13:1026064.
doi: 10.3389/fphar.2022.1026064. eCollection 2022.

Editorial: Prevention, diagnosis and treatment of rare disorders

Affiliations
Editorial

Editorial: Prevention, diagnosis and treatment of rare disorders

Timothy M Cox et al. Front Pharmacol. .
No abstract available

Keywords: diagnosis; equitable access; global; orphan drugs; prevention; rare diseases; treatment; venture capitalism.

PubMed Disclaimer

Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Comment on

  • Editorial on the Research Topic Prevention, diagnosis and treatment of rare disorders

References

    1. Bill & Melinda Gates foundation (2022). We are a nonprofit fighting poverty, disease and inequity around the world. Available at: https://www.gatesfoundation.org/ (Accessed Aug. 21, 2022).
    1. Drelichman G., Castañeda-Hernández G., Cem Ar M., Dragosky M., Garcia R., Lee H., et al. (2020). The road to biosimilars in rare diseases - ongoing lessons from Gaucher disease. Am. J. Hematol. 95 (3), 233–237. 10.1002/ajh.25701 - DOI - PMC - PubMed
    1. European Medicines Agency (2000). Legal framework: Orphan designation. Available at: https://www.ema.europa.eu/en/human-regulatory/overview/orphan-designatio... .
    1. Koestler A. (1941). Darkness at noon. Translated by Daphne Hardy. New York: Modern Library Books, MacMillan Co.
    1. Matalonga L., Laurie S., Papakonstantinou A., Piscia D., Mereu E., Bullich G., et al. (2020). Improved Diagnosis of rare disease patients through systematic detection of runs of homozygosity. J. Mol. Diagn. 22 (9), 1205–1215. 10.1016/j.jmoldx.2020.06.008 - DOI - PMC - PubMed

Publication types

LinkOut - more resources