The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…
CB 2679d-GT, an investigational gene therapy for hemophilia B being developed by Catalyst Biosciences, can increase factor IX (FIX) levels and significantly reduce bleeding, early studies show. The data, “Combination of a Novel Chimeric AAV Capsid and Potency Enhanced FIX Variant for Hemophilia B…
The European Medicines Agency (EMA) has approved 21-day dosing intervals for CSL Behring‘s Idelvion (rIX‐FP) as a prophylactic treatment for adults with hemophilia B. Idelvion — which is…
People with hemophilia A in the U.K. will soon have greater access to Elocta (efmoroctocog alfa) under a two-year agreement between Sobi and the National Health Service (NHS), the company announced. Elocta was approved by the European Commission for the treatment and prevention of bleeding in all…
Sobi and Sanofi announced an additional donation of up to 500 million international units (IUs) of clotting factor therapy in support of the World Federation of Hemophilia‘s Humanitarian Aid Program. The clotting factor goes toward treating people with hemophilia in developing countries where access to medicine is…
CSL Behring has acquired exclusive global rights to commercialize AMT-061, a potential gene therapy for hemophilia B now in a pivotal clinical trial, from its developer uniQure. Under terms of the agreement, uniQure will receive an upfront payment of $450 million from CSL Behring, and is eligible…
Valoctocogene roxaparvovec, BioMarin Pharmaceutical‘s investigational gene therapy, results in a more than 90% drop in annual bleeding rates and the use of clotting factor VIII (FVIII) in men with severe hemophilia A, updated data from a Phase 1/2 clinical trial show. It also precluded the need…
The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…
As a long-term preventive treatment, fitusiran leads to sustained reductions in bleed frequency in people with moderate-to-severe hemophilia A or B regardless of inhibitor status, early results from an extension study show. The findings were presented at the recent World Federation of Hemophilia Virtual Summit and…
Up to 14 months after a one-time infusion of SB-525, an investigational gene therapy, durable increases in the activity of clotting factor VIII (FVIII) are continuing in all five severe hemophilia A patients treated at the highest dose in the Alta Phase 1/2 trial. None of these…
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