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- Title
Does the orphan medicinal product regulation assist or hinder access to innovative haemophilia treatment in Europe?
- Authors
O'Mahony, B.; Peyvandi, F.; Bok, A.
- Abstract
The author discusses the effects of the orphan medicinal product regulation on the development of innovative hemophilia treatments in Europe. Topics covered include the marketing authorization offered to acting factor IX treatment products in the U.S. in 2014. Also mentioned is the use of prophylaxis to lessen the bleeding risks of patients with hemophilia.
- Subjects
HEMOPHILIA treatment; BLOOD coagulation factor IX; ORPHAN drugs; HEMOPHILIACS; HEMORRHAGE risk factors; DRUG therapy
- Publication
Haemophilia, 2014, Vol 20, Issue 4, p455
- ISSN
1351-8216
- Publication type
Editorial
- DOI
10.1111/hae.12462