News

BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec continues to safely and effectively prevent bleeding episodes and the need for prophylactic clotting factor VIII in adults with severe hemophilia A, four-year data from a Phase 1/2 clinical trial show. The results were submitted for presentation at the World Federation…

A new type of gene therapy that uses RNA instead of DNA led to rapid and prolonged production of therapeutic levels of factor VIII (FVIII) in a mouse model of hemophilia A, a study reported. The study, “Treatment of…

Infection by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the coronavirus causing COVID-19, can induce the onset of acquired hemophilia A (AHA), a case report has found. As such, blood coagulation should be monitored closely in patients hospitalized for COVID-19, scientists said. The study, “…

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…

The U.S. Food and Drug Administration (FDA) has approved Expression Therapeutics‘ request to open a Phase 1 trial of ET3, an investigational gene therapy for people with hemophilia A. The decision followed a review of the company’s investigational new drug application (IND) requesting clearance of ET3…

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…

Novo Nordisk has issued a notice warning healthcare professionals that the hemophilia B treatment Rebinyn (nonacog beta pegol, or N9-GP) can interfere with some laboratory tests that measure blood coagulation. Following reports that people with severe COVID-19 may develop a blood-clotting condition similar to…

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s son, Maximilian, has cystic fibrosis (CF), making him particularly susceptible to COVID-19 and the complications that can arise from it.

Long-term treatment with Rebinyn (nonacog beta pegol, or N9-GP) is safe and effective at preventing and treating spontaneous bleeds in previously treated children, 12 or younger, with severe hemophilia B, five-year data from a Phase 3 trial show. The findings were reported in a study, “…

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…