FDA official calls UniQure's gene therapy a 'failed' treatment for Huntington's disease
Teona Gherasim
UniQure needs to run another study to prove that its gene therapy "actually helps people with Huntington's disease," a senior U.S. Food and Drug Administration official said on a call with reporters Thursday. The official, who requested anonymity before discussing sensitive information, confirmed the agency has asked the company to run a placebo controlled trial of its treatment, which is administered directly into the brain. UniQure has said that type of study isn't ethical because it would
din zilele anterioare