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669,673 Results for "world federation of hemophilia".
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Approvals
Sanofi, Alnylam Win First RNAi Approval for Hemophilia A and B
Unlike other hemophilia therapies, Qfitlia is indicated for both hemophilia A and B, and can be given in patients regardless of the presence of neutralizing antibodies against clotting factor VIII or IX.
March 31, 2025
·
2 min read
·
Tristan Manalac
Gene therapy
Pfizer Axes Beqvez Worldwide, Pivoting Away From Gene Therapy for Hemophilia
The move comes weeks after Pfizer terminated its partnership with Sangamo Therapeutics for another hemophilia gene therapy.
February 21, 2025
·
2 min read
·
Tristan Manalac
Rare diseases
Novo’s Bispecific for Hemophilia Aces Phase III Pediatric Trial
Novo Nordisk’s bispecific antibody Mim8 prevented bleeding events and caused no adverse safety events in the Phase III FRONTIER3 trial. The company plans to file for approval this year, hoping to compete in the hemophilia A space with Roche’s
blockbuster
Hemlibra.
February 7, 2025
·
2 min read
·
Dan Samorodnitsky
Genetown
hC Bioscience Announces Lead Program in Hemophilia and Reports Positive Preclinical Data on Novel Protein Editing Approach Using Anticodon Engineered tRNA
hC Bioscience today announced preclinical data supporting its lead program in severe hemophilia A at the World Federation of Hemophilia 2024 World Congress in Madrid, Spain.
April 23, 2024
·
2 min read
Press Releases
FDA Approves Qfitlia™ (fitusiran), the First siRNA (RNAi Therapeutic) for the Treatment of Hemophilia A or B
March 31, 2025
·
7 min read
Press Releases
Children’s Hospital of Philadelphia Researchers Find Gene Therapy Shows Long-Term Benefits for Hemophilia B Patients
April 18, 2025
·
4 min read
Government
Appeals Court Stops Rehiring of Fired Federal Workers, Including From HHS
The Fourth Circuit’s ruling follows a Supreme Court verdict that also allowed the Trump administration to move forward with its mass layoffs at federal agencies.
April 10, 2025
·
2 min read
·
Tristan Manalac
Press Releases
Press Release: Qfitlia approved as the first therapy in the US to treat hemophilia A or B with or without inhibitors
March 31, 2025
·
9 min read
Approvals
Pfizer Wins FDA Nod for Hemophilia Therapy Amid Activist Investor Challenge
The approval makes Pfizer’s Hympavzi the first once-weekly subcutaneous prophylactic injection for hemophilia B in the U.S., according to the company, which is currently embroiled in a row with activist investor Starboard Value.
October 14, 2024
·
2 min read
·
Tristan Manalac
Press Releases
FDA Approves Novel Treatment for Hemophilia A or B, with or without Factor Inhibitors
March 28, 2025
·
4 min read
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