News

February 19, 2021February 19, 2021

Efanesoctocog Alfa on Fast Track for Hemophilia A

News

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 ... Read more

February 18, 2021February 18, 2021

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

News, syndicated

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 ... Read more

February 17, 2021February 17, 2021

Hemlibra Can Help Manage Bleeding in AHA, Study Finds

News

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 ... Read more

February 15, 2021February 15, 2021

Preventive Advate Found Better Than On-demand Treatment in Hemophilia A

News

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 ... Read more

February 12, 2021February 12, 2021

Nasal Desmopressin Recall Affects Availability Through 2023

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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 ... Read more

February 10, 2021February 11, 2021

Pandemic Won’t Stop Rare Disease Day on Feb. 28

News, syndicated

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 ... Read more

February 8, 2021February 11, 2021

Gene Therapy SPK-8016 Shows Early Promise for Severe Hemophilia A

News

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 ... Read more

February 5, 2021February 5, 2021

MarzAA Shows Promise in Hemophilia Animal Models

News

The investigational medication marzeptacog alfa activated (MarzAA) showed promise at stopping bleeding in animal models of hemophilia. The findings were presented in two posters at the Annual Congress of the ... Read more

February 3, 2021February 3, 2021

Kovaltry Safe, Effective for Children With Hemophilia A in Real-world Study

News

Kovaltry (octocog alfa) is effective and safe as a preventive treatment for children with hemophilia A, a European real-world study reports. The study, “Real-world evidence on Kovaltry (81-8973) in children ... Read more

February 2, 2021February 2, 2021

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

News, syndicated

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs ... Read more