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
. 2016 May;109(5):361.
doi: 10.1093/qjmed/hcv150. Epub 2015 Sep 3.

Orphan drugs: expensive yet necessary

H I Hyry  1 , J C Roos  2 , T M Cox  2
Affiliations

Orphan drugs: expensive yet necessary

H I Hyry et al. QJM. 2016 May.
No abstract available

PubMed Disclaimer

References

    1. Hyry HI, Roos JCP, Cox TM. Orphan drugs: expensive yet necessary. QJM 2015; 108:269–72. - PubMed
    1. Hyry HI, Stern AD, Cox TM, Roos JCP. Limits on use of health economic assessments for rare diseases. QJM 2014; 107:241–5. - PubMed
    1. Hyry HI, Roos JCP, Manuel J, Cox TM. The legal imperative for treating rare disorders. Orphanet J Rare Dis 2013; 8:135. - PMC - PubMed
    1. Müller-Hill B. Murderous Science: Elimination by Scientific Selection of Jews, Gypsies, and Others in Germany 1933-45. Oxford, Oxford University Press, 1998.
    1. Andersson H, Kaplan P, Kacena K, Yee J. Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1. Pediatrics 2008; 122:1182–90. - PubMed