Record number of medicines for rare diseases recommended for approval in 2014
Number of medicines with new active substances continues to increase
13-Jan-2015 -
In 2014, the European Medicines Agency (EMA) recommended the highest number of orphan designated medicines for marketing authorisation in a year. Out of the 82 medicines for human use recommended in 2014, 17 are intended for the treatment of a rare disease, providing therapies for patients who ...
Daklinza
Duchenne muscular dystrophy
erythropoietic protoporphyria
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