With 2016 numbers now available, the number of orphan drugs in development for neurological indications is looking quite positive. I have reviewed the numbers of orphan drug designations and approvals by FDA in 2016 to see how popular are neurological orphan drugs today and what the trend is for the near future.
The journal Nature just released one of the most anticipated breaking news of the last few years: CRISPR gene editing has been tested in a person for the first time. In my day-to-day work I interact with families that have a child with a genetic disease. I get one question a lot: how close are we to turn that discovery into a therapy for people with genetic diseases?