Why we should care about ultra-rare disease

Author:

Harari Sergio

Publisher

European Respiratory Society (ERS)

Subject

Pulmonary and Respiratory Medicine

Reference20 articles.

1. OrphaNet. www.orpha.net/consor/cgi-bin/index.php?lng=EN Date last updated: March 10, 2016. Date last accessed: May, 2015.

2. A journey of hope: lessons learned from studies on rare diseases and orphan drugs

3. Rare (pulmonary) disease day: “feeding the breath, energy for life!”

4. National Institute for Clinical Excellence. NICE Citizens Council Report Ultra Orphan Drugs. London, NICE, 2004.

5. Drugs for exceptionally rare diseases: do they deserve special status for funding?

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