Hemophilia Need Not Be Severe to Affect Life Quality for Men and Women
Mild-to-moderate hemophilia has a significant impact on the physical and psychosocial health, and life quality, of both men and…
Articles by Marta Figueiredo, PhD
Roctavian, Potential Hemophilia A Gene Therapy, Gets Fresh FDA Support
The U.S. Food and Drug Administration (FDA) has given regenerative medicine advanced therapy (RMAT) designation to Roctavian, an investigational…
Factor VII Treatment Eptacog Beta Now Under Review in Europe
The European Medicines Agency (EMA) has agreed to review an application by Laboratoire Francais du Fractionnement et des Biotechnologies, a…
Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of…
Voluntary Recall Issued for Batch of Mononine, Hemophilia B Treatment
CSL Behring has issued a voluntary, pharmacy level recall covering one batch of Mononine, its long-standing treatment for…
Optimal Disease Control Advised for Hemophilia B Patients
People with severe hemophilia B are likely to develop hemophilic arthropathy — a painful and degenerative joint disease caused by…
uniQure’s Hemophilia B Gene Therapy Program Placed on Hold
The U.S. Food and Drug Administration (FDA) has placed a clinical hold on uniQure’s gene therapy program for hemophilia…
High Dose of Gene Therapy SB-525 Raises FVIII Levels, Prevents Bleeds, Alta Trial Data Show
When administered at the highest dose, the investigational gene therapy SB-525 (giroctocogene fitelparvovec) prevented bleeds and the use of…
Gene Therapy AMT-060 for Hemophilia B Prevents Bleeds Up to 5 Years, Ongoing Trial Shows
AMT-060, a first-generation gene therapy candidate by uniQure, continues to safely reduce the number of bleeding episodes and…
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe
The first wave of COVID-19Â in Europe severely disrupted access to care and raised stress and anxiety in people with…