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February 17, 2020February 17, 2020

Light Chain, Takeda Reach Key Milestone in Partnership Developing Bispecific Antibodies for Treating Hemophilia A

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Light Chain Bioscience, a unit of Swiss biotech Novimmune, has completed the discovery phase of its collaborative program with Takeda for developing bispecific antibodies — protective proteins produced by ... Read more

February 14, 2020February 17, 2020

High Dose of Gene Therapy for Hemophilia B Normalizing FIX Levels, Early Trial Data Show

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When administered at a high dose, a gene therapy for hemophilia B known as FLT180a seems able to restore the activity of clotting factor IX (FIX) within a normal range in men ... Read more

February 12, 2020February 12, 2020

Esperoct Available in US to Treat Adults and Children with Hemophilia A, Novo Nordisk Says

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Esperoct (turoctocog alfa pegol) is now available in the U.S. to treat and control bleeding in adults and children with hemophilia A, the therapy’s manufacturer, Novo Nordisk, announced. The treatment’s ... Read more

February 10, 2020February 10, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

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The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous ... Read more

February 10, 2020February 10, 2020

Catalyst Presents Positive Data of Under-the-Skin Candidate Therapies for Preventing, Treating Bleeds

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Catalyst Biosciences has announced new clinical data supporting the safety and efficacy of its lead candidate therapies for treating hemophilia. Factor IX (FIX) dalcinonacog alfa (DalcA) was found to ... Read more

February 7, 2020February 14, 2020

MRI Superior to Radiography in Capturing Joint Changes That Signal Future Bleeds, Study Says

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Joint changes detected by magnetic resonance imaging (MRI), and often are missed on radiographs, are a strong predictor of damaging bleeds over the next five years in joints of ... Read more

February 5, 2020February 5, 2020

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

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The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over ... Read more

February 5, 2020February 5, 2020

Researchers Develop Faster, Less Expensive, Non-invasive Method for Hemophilia A Prenatal Diagnosis

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Researchers have developed a faster, less expensive, reliable method for non-invasive prenatal diagnosis (NIPD) of hemophilia A. Future, larger studies are required to confirm the utility of the method, ... Read more

February 3, 2020February 3, 2020

First Child with Hemophilia B Dosed in Phase 4 Trial to Extend Ixinity to Those Under Age 12

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Aptevo Therapeutics announced the dosing of a first patient in a Phase 4 study aiming to extend use of its prophylactic (preventive) treatment Ixinity (coagulation factor IX ) to ... Read more

January 31, 2020January 31, 2020

coreHEM Results Lead to Joint Effort to Create Tool for Assessing Mental Health in Gene Therapy

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A publicly available, patient-reported outcome measure is being jointly developed by Green Park Collaborative (GPC) and the National Hemophilia Foundation (NHF) to evaluate the effect of gene therapy on the ... Read more

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