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UPCOMING EVENTS

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BioMarin recently announced positive updates from its ongoing phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. The trial currently includes 134 participants, all of whom have received a single dose of the therapy and have been observed for at least one year post treatment.

CSL Behring has announced a voluntary pharmacy level product recall of one batch of Mononine Coagulation Factor IX (Human), a plasma-derived product used to treat hemophilia B. CSL Behring’s notification states, “Patients can continue to use product they may have. Although the potential for safety risk to patients is considered low, it cannot be fully excluded.”

The affected batch is identified as: P100125501

The new joint clinical practice guidelines on the diagnosis and management of von Willebrand disease, developed by the American Society of Hematology (ASH), the International Society of Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF), and the World Federation of Hemophilia (WFH) now have snapshot guides available in English, Spanish, and French.

The snapshots provide a high-level summary of both the diagnosis and management of VWD.

 

The new joint clinical practice guidelines on the diagnosis and management of von Willebrand disease, developed by the American Society of Hematology (ASH), the International Society of Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF), and the World Federation of Hemophilia (WFH) now have snapshot guides available in English, Spanish, and French.

The snapshots provide a high-level summary of both the diagnosis and management of VWD.

 

“From Principles to Practice: Preparing for the Advent of Gene Therapy” represents the newest educational activity in Medscape’s series “Clinical Advances in Gene Therapy for Hemophilia.” Presented through a collaboration between Medscape and the National Hemophilia Foundation, the series includes various discussions relevant to the latest data on gene therapy as well as the practicalities associated with using gene therapy for hemophilia.

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