uniQure Gene Therapy AMT-060 Shows Long-term Efficacy in Hemophilia B Patients
Gene therapy company uniQure released new data showing that its lead therapy AMT-060 for severe and moderately severe hemophilia B is…
Articles by Patricia Inácio, PhD
Fitusiran Clinical Development Among 2018 Corporate Goals, Alnylam Pharmaceuticals Says
Among its corporate goals for 2018 announced recently, Alnylam Pharmaceuticals said it will continue to clinically develop the investigational RNA therapy …
Clinical Trial Testing CB 2679d in Hemophilia B Patients is Shortened
Catalyst Biosciences has amended the protocol of its ongoing Phase 1/2 clinical trial investigating the highly potent recombinant human factor IX…
FDA Lifts Clinical Hold on Investigational Therapy Fitusiran
The U.S. Food and Drug Administration (FDA) has lifted the hold on clinical studies with fitusiran, an investigational therapy…
New Analysis Confirms FDA-approved Alprolix Benefits in Patients with Severe Hemophilia B
A new analysis of two Phase 3 clinical trials confirms that extending preventive treatment with Alprolix is safe and leads…
Genentech’s Hemlibra Maintains Strong Bleeding Control Results in Phase 3 Trial, Interim Analysis Shows
Injection of Hemlibra (emicizumab-KXWH) every four weeks helps control bleeding in hemophilia A patients, an interim analysis…
First Hemophilia A Patient Enrolls in Phase 4 Trial for Eloctate Immune Tolerance Induction
Swedish Orphan Biovitrum (Sobi) announced the successful enrollment of the first hemophilia A patient in the company’s ReITIrate clinical trial.
Pfizer Debuts Innovative Video Game and Wristband for Children with Hemophilia
Pfizer Rare Disease has developed a new video game and wearable wristband to help hemophilia patients learn more about their…
Emicizumab, Genentech’s Therapy for Hemophilia A, Granted Priority Review by the FDA
The U.S. Food and Drug Administration (FDA) has granted priority review to the therapy emicizumab for patients with hemophilia A…
Patient Surveys Can Help Doctors Do a Better Job of Treating Hemophilia, Study Suggests
Information from patient surveys can help doctors do a better job of managing hemophilia and improving patients’ outcomes, an American…