ALLMedicine™ Hemophilia B Center

Mar 25th, 2023 - Therapy for hemophilia B is aimed at replacing the congenital deficiency of coagulation factor IX (FIX). For replacement therapy, several FIX concentrates derived from donated human plasma or engineered by recombinant DNA technology are currently ...

Mar 17th, 2023 - AN OPEN-LABEL, NON-INVESTIGATIONAL PRODUCT, MULTI-CENTER, LEAD-IN STUDY TO EVALUATE PROSPECTIVE EFFICACY AND SELECTED SAFETY DATA OF CURRENT FACTOR IX (FIX) OR FACTOR VIII (FVIII) PROPHYLAXIS REPLACEMENT THERAPY IN THE USUAL CARE SETTING OF MODERA...

Mar 6th, 2023 - This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-a...

Mar 3rd, 2023 - Evaluation of the long-term level of persistence and potential late or delayed adverse events associated with PF-06838435 (formerly SPK-9001), assessment of the durability of the transgene expression, and determination of the effects of PF-0683843...

Feb 28th, 2023 - Long-term efficacy and safety of the extended half-life recombinant FIX Fc fusion protein (rFIXFc) has been established in previously treated patients with severe hemophilia B in 2 Phase 3 trials (B-LONG [NCT01027364] and Kids B-LONG [NCT01440946]...

Mar 17th, 2023 - AN OPEN-LABEL, NON-INVESTIGATIONAL PRODUCT, MULTI-CENTER, LEAD-IN STUDY TO EVALUATE PROSPECTIVE EFFICACY AND SELECTED SAFETY DATA OF CURRENT FACTOR IX (FIX) OR FACTOR VIII (FVIII) PROPHYLAXIS REPLACEMENT THERAPY IN THE USUAL CARE SETTING OF MODERA...

Mar 6th, 2023 - This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-a...

Mar 3rd, 2023 - Evaluation of the long-term level of persistence and potential late or delayed adverse events associated with PF-06838435 (formerly SPK-9001), assessment of the durability of the transgene expression, and determination of the effects of PF-0683843...

Feb 24th, 2023 - Study B7841007 is an open-label extension study to assess the long-term safety, tolerability, and efficacy of prophylaxis treatment with marstacimab in participants who did not require "Early Termination" from the Phase 3 Study B7841005. Approxima...

Feb 22nd, 2023 - Hemophilia A is a condition in which blood is unable to clot effectively. It is caused by a mutation or deletion in the gene that is responsible for producing blood-clotting factor VIII protein. Individuals with hemophilia A suffer from repeated b...

Dec 12th, 2022 - NEW ORLEANS — A gene therapy recently made headlines for becoming the most expensive pharmaceutical ever launched — the price tag was $3.5 million for a one-off treatment with etranacogene dezaparvovec (Hemgenix) for hemophilia B. A gene therapy f...

Nov 23rd, 2022 - The U. S.

Nov 23rd, 2022 - The U.S. Food and Drug Administration has approved etranacogene dezaparvovec (Hemgenix), the first gene therapy option for adults with hemophilia B who currently use factor IX prophylaxis therapy, have current or historical life-threatening hemorr...

Nov 22nd, 2022 - Nov 22 (Reuters) - The U.S. health regulator on Tuesday approved Australian drugmaker CSL Ltd and partner Uniqure NV’s gene therapy for hemophilia B, potentially offering a long-term solution for patients with the blood clotting disorder. The appr...

Nov 22nd, 2022 - The FDA on Tuesday approved the first gene therapy for treating hemophilia B, a genetic bleeding disorder resulting from missing or insufficient levels of factor IX. Etranacogene dezaparvovec (Hemgenix) is indicated for adults with the condition w...