Posted by Michael Wonder on 19 Sep 2018
Can patient advocates help make Akcea the next Sarepta and overcome the FDA’s rejection?
18 September 2018 - Patients with a rare disease are asking the FDA a question the agency rarely hears: Is that really your final answer?
A group for people with familial chylomicronemia syndrome, a genetic condition that causes fat to build up in a person’s blood, is circulating a petition that begs the FDA to reconsider its decision to reject the one experimental treatment for FCS, Akcea’s volanesorsen. As of Monday, the petition had more than 8,000 signatures.
