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BioMarin, following sluggish sales, to offload hemophilia gene therapy
27 Oct 2025 23:49 GMT
… is giving up on the hemophilia gene therapy Roctavian, announcing … the chronic therapies people with hemophilia A use to prevent bleeding … therapy for the less common hemophilia B. Uptake has also … for CSL’s Hemgenix, another hemophilia B gene therapy.
Last …
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UK MHRA grants marketing authorization to Novo Nordisk's concizumab to prevent or reduce the frequency of bleeding episodes in people aged 12 years and older with haemophilia A or B with inhibitors
27 Oct 2025 06:29 GMT
… years and older with haemophilia A or B with … (clotting factor VIII for haemophilia A and clotting factor IX … for haemophilia B).
The new marketing … prevent bleeding in people with haemophilia.?
Concizumab is injected subcutaneously …
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BioMarin Pharma plans to divest struggling gene therapy
27 Oct 2025 23:36 GMT
… to treat adults with severe hemophilia A, a rare genetic disorder …
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BioMarin Reports Third Quarter 2025 Results and Provides Corporate Update
27 Oct 2025 20:32 GMT
… play in the treatment of hemophilia A and are therefore evaluating …
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H.C. Wainwright Initiates Rani Therapeutics (RANI) with Buy and $11 PT on RaniPill Potential
27 Oct 2025 19:14 GMT
… with Chugai Pharmaceutical for a hemophilia product, and an additional five …
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Hemab collects another $157M to go after ‘underserved’ blood diseases
27 Oct 2025 16:06 GMT
… said.
Dive Insight:
Innovation in hemophilia has taken off over the … on newer approaches. As with hemophilia, they’re aiming for better …
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UK NHS Walks Back First-Cousin Marriage
27 Oct 2025 12:29 GMT
… disease, thalassemia, congenital heart defects, hemophilia A, metabolic disorders, and others …
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Clear Cell Ovarian Carcinoma and Its Distinct Coagulopathy Profile: Molecular Drivers and Clinical Implications
27 Oct 2025 12:16 GMT
… factor pathway inhibitor: implications for hemophilia therapy. J Thromb Haemost. 2022 …
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Hemab Therapeutics Announces $157 Million Series C Financing to Advance Next-Generation Treatments for Underserved Bleeding Disorders
27 Oct 2025 10:42 GMT
… as breakthrough prophylactic therapies transformed hemophilia care," said Jeanette Cesta … has already changed lives in hemophilia."
Patients' Insights Drive …
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CSL Honoured as Overall Winner of the 2025 Facility of the Year Awards by the International Society for Pharmaceutical Engineering
27 Oct 2025 08:36 GMT
… medicines, including those that treat haemophilia and immune deficiencies, vaccines to …