News

Late Replacement Therapy Tied to Poor Outcomes Following Bleeds

Serious bleeds are often a fatal complication in people with hemophilia if not managed early, a 10-year study from Senegal shows. These findings highlight the need for prompt diagnosis of severe bleeding and early administration of replacement therapy to lower the risk of mortality. In resource-limited countries, increasing patients’ and…

World Hemophilia Day Supporters Hope to Make a Difference

Supporters of World Hemophilia Day, on April 17, are hoping to raise awareness about the bleeding disorder with events planned globally and on social media, and to advocate for sustainable and equitable access to care and treatment. The annual event, which is organized by the World Federation of Hemophilia…

EtranaDez Under Review in Europe as 1st Gene Therapy for Hem B

The European Medicines Agency (EMA) has agreed to review a request by CSL Behring to approve the potential gene therapy EtranaDez (etranacogene dezaparvovec) for people with hemophilia B. The request, in the form of a marketing authorization application or MAA, will be reviewed under the EMA’s accelerated assessment, meaning…

Increased Education Crucial to Improving Rare Disease Care, Survey Finds

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…