A personal perspective of orphan drug development for rare diseases: A golden opportunity or an unsustainable future?

Oo, C. and Rusch, L. M. Journal of Clinical Pharma. doi: 10.1002/jcph.599

Nevertheless, there is no better opportunity than the present to develop orphan drugs in order to accelerate the treatment and alleviate the suffering of patients with rare diseases.


 A personal perspective of orphan drug development for rare diseases: A golden opportunity or an unsustainable future?