Effectiveness in controlling bleeds and the potential for less frequent use of a treatment are two priorities people with hemophilia place on a potential gene therapy, and both ranked slightly higher than safety, a survey study of patients in the U.S. reports. The study, “Patient preferences and…

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…

In the Netherlands, children and adults with hemophilia participate in sports as much or more than people from the general population, according to a study. The findings also indicated that disease severity affected sports participation only in adults, and that children with hemophilia were more willing to participate in…

A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 safely and effectively prevented bleeding episodes and the use of clotting factor VIII (FVIII) for up to four years in men with hemophilia A, according to the latest data from a Phase 1/2 trial and its extension study.