Treatment with Octapharma‘s Nuwiq is associated with a low risk of developing inhibitors — antibodies that prevent the medication from working properly — in previously untreated patients (PUPs) with hemophilia A, according to the results of a clinical trial. These findings were published in the journal Thrombosis…
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Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…
Exercises to control posture could improve joint health in children with hemophilia and should form part of regular rehabilitation programs, a study suggests. The balance disturbances uncovered in the study could provide early signs of musculoskeletal disorders that could be prevented or reduced through prompt diagnosis and therapy, researchers…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to efanesoctocog alfa, an investigational factor VIII (FVIII) replacement therapy for treating hemophilia A, Sanofi, one of the therapy’s developers, announced in a press release. This designation is given to accelerate the development and review of medications…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…
Treatment with Hemlibra (emicizumab) can help to effectively manage bleeding in individuals with acquired hemophilia A (AHA), according to a new study. While clinical trials will need to be done to fully assess Hemlibra as an AHA therapy, the researchers said this medication also has the potential to…
When given as a prophylactic, or preventive therapy, Advate worked better than on-demand treatment at lowering the number of spontaneous bleeds in people with moderate or severe hemophilia A, according to seven-year data from a real-world study. A separate analysis of the study, called AHEAD International (NCT02078427),…
An internal investigation conducted by Ferring Pharmaceuticals into its recently recalled desmopressin nasal spray formulations found an issue with the tightness of the bottle seal. Measures taken to prevent recurrence of the issue will affect the availability of the products through at least 2023.
Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
A single dose of the experimental gene therapy SPK-8016 showed promising safety and efficacy in lowering the frequency of bleeds and the need for infusions in men with severe hemophilia A. These preliminary findings from an ongoing Phase 1/2 trial (NCT03734588) were presented by Spencer Sullivan, MD,…
