Search Results for: hemophilia

May 15, 2020May 15, 2020

Hemophilia A Gene Therapy, AMT-180, Continues to Show Activity, Safety in Animals

News

A single dose of the experimental gene therapy AMT-180 continues to be well-tolerated and to enhance blood clotting activity independent of factor VIII levels in mouse and primate models ... Read more

May 13, 2020May 13, 2020

BioMarin’s Gene Therapy for Hemophilia A Remains on Track for US Approval

News

Despite the COVID-19 pandemic, BioMarin Pharmaceutical‘s application to the U.S. Food and Drug Administration (FDA) seeking approval of valoctocogene roxaparvovec, an experimental gene therapy for adults with severe hemophilia A, ... Read more

May 11, 2020May 11, 2020

Risk of Bleeds in Hemophilia A Influenced by Treatment History, Other Factors, Study Suggests

News

The risk of spontaneous bleeding episodes in patients with severe hemophilia A receiving preventive treatment changes over time and can be influenced by treatment history and other patient-related factors, ... Read more

May 8, 2020May 8, 2020

Generic Version of DDAVP to Treat Mild Hemophilia A Launches in US

News

Dr. Reddy’s Laboratories announced the U.S. launch of a generic version of DDAVP (desmopressin acetate), used to control bleeding in people with mild hemophilia A. The generic is available under the ... Read more

May 5, 2020May 1, 2020

Protecting Hemophiliacs from the Unknowns of COVID-19

Columns, The Forgotten Factor - a Column by Shellye Horowitz

Our hemophilia community has been here before. A novel virus — previously, it was HIV — hit the world, and our community was uncertain about how it would affect ... Read more

May 4, 2020May 4, 2020

Prophylaxis Better Than On-demand Therapy for Reducing Bleeds in Hemophilia A, Study Reports

News

Switching from on-demand to preventive treatment — or prophylaxis — significantly reduces bleeding in adults with hemophilia A, including the elderly, a real-life study shows. The study, “Long‐term analysis ... Read more

May 1, 2020May 1, 2020

Catalyst Biosciences Awarded EU Patent for Experimental Hemophilia B Therapies

News

Catalyst Biosciences has received a European Union patent for its investigational hemophilia B treatments, including its lead therapy candidate, dalcinonacog alfa (DalcA) and a preclinical gene therapy candidate called ... Read more

April 29, 2020April 29, 2020

Awareness Key to Proper Treatment Adherence in Hemophilia, Study Says

News

Greater awareness of hemophilia and its symptoms is associated with better adherence to treatment by patients, and those with more severe disease are most likely to use a treatment as ... Read more

April 27, 2020April 27, 2020

2 Proteins May Help Predict Acute Joint Bleeding in Severe Hemophilia A, Study Suggests

News

Blood levels of two proteins — C-reactive protein (CRP) and vascular endothelial growth factor (VEGF) — involved in inflammation and blood vessel formation are significantly associated with, and may ... Read more

April 22, 2020April 22, 2020

DalcA Effective and Safe as Hemophilia B Injection Treatment, Study Suggests

News

Data from a completed Phase 2b trial indicate that dalcinonacog alfa (DalcA) is an effective and safe therapy to prevent bleeding in people with severe hemophilia B. Final results from ... Read more

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