Patients with a rare disease are asking the Food and Drug Administration a question the agency rarely hears: Is that really your final answer?
A group for people with familial chylomicronemia syndrome (FCS), 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.
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