FDA hemophilia meeting to discuss new efficacy endpoints

FDA hemophilia meeting to discuss new efficacy endpoints

Source: 
BioCentury
snippet: 

FDA will hold a public workshop on Dec. 6 to discuss development of therapies for hemophilia, including new efficacy endpoints. The agency plans to develop patient-reported outcomes for hemophilia trials and discuss pediatric enrollment, long-term safety assessments and measurements for factor VIII and IX levels in gene therapy trials.