What Was the Most Notable Orphan Drug Approval in 2017?

Gilbert: Well, it’s interesting because there are many orphan drugs that have been approved and are in the pipeline, but for a patient who has an orphan or rare disease, whichever one is treating their disease is the most notable.

Many of these patients have not had treatment options before. So, the most important one is the one that is treating you, as the patient, or your family member.