Tags » Hemophilia A

BioMarin to Begin Phase 3 Gene Therapy Trials for Hemophilia A

BioMarin announced the results from their ongoing Phase 1/2 gene therapy studies evaluating  BMN 270 for patients with hemophilia A. Based on encouraging data, Phase 3 studies are being planned to start later this year. 433 more words


EU Grants €5.6 Million to Consortium Developing Sernova’s Cell Pouch Therapy for Hemophilia A Patients

The European Commission will award the Brussels-based HemAcure Consortium €5.6 million in next-stage funding of a therapy called Cell Pouch —  developed in collaboration with Canada’s  291 more words


Phase 3 Trials Evaluating Further Potential of 2 Treatments for Hemophilia A

Interim results of Phase 3 clinical studies investigating two major challenges in hemophilia — the development of inhibitors (antibodies to the drug used to treat bleeding episodes) and the need for repeated venous injection of blood clotting protein Factor VIII (FVIII) — were recently released. 543 more words


FDA Accepts Bioverativ’s Investigational New Drug Application for BIVV001 to Treat Hemophilia A

The U.S. Food and Drug Administration (FDA) has accepted Bioverativ’s investigational new drug application (IND) for BIVV001, an investigational Factor VIII therapy designed to extend protection from bleeds in  302 more words


New Project Aims to Set Effectiveness Guidelines for Hemophilia Gene Therapy Trials

An international team has joined efforts to establish guidelines for effectiveness and outcome measurements regarding gene therapies in hemophilia.

The CoreHEM project will be led by researchers from  492 more words


The grief process.

After the diagnosis, Alaina’s factor VIII levels needed to go from <1% to 100% in order to go through with the craniotomy. Despite the surgery, the damage was too severe. 464 more words