Articles related to HEMOPHILIA

Valoctocogene roxaparvovec gene therapy demonstrates long-term efficacy in reducing bleeding episodes and factor VIII use in patients with severe hemophilia A, potentially changing the treatment landscape for this condition.

Roctavian represents a turning point in hemophilia A management, offering patients like Maxwell a potential lifetime of relief from traditional therapies. This gene therapy directly targets the genetic cause, paving the way for innovative treatments in genetic disorders.

This UK study highlights the urgent need for targeted interventions and close monitoring to improve the quality of life for hemophilia patients suffering from ankle arthropathy.

The business launched a precautionary global recall of desmopressin formulations used to treat mild-to-moderate hemophilia A and von Willebrand disease after higher-than-specified amounts of desmopressin were discovered in various formulations. Stimate, a nasal spray containing a synthetic form of desmopressin acetate used to control bleeding in hemophilia patients, is among the recalled products, as is Octostim Nasal Spray 1.5 mg/ml, DDAVP 10 micrograms (mcg)/0.1 ml, Minirin Nasal Spray 0.1 mg/ml, and Generic Desmopressin Acetate Nasal Spray 10 mcg/0.1 ml. Products containing higher-than-specified amounts of desmopressin were recalled due to concerns that increased desmopressin exposure can result in water retention, low blood pressure, and low blood salt levels in mild cases, as well as seizure, coma, and death.

These evidence-based guidelines from the American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF), and the World Federation of Hemophilia (WFH) are intended to support patients, clinicians, and health care professionals in their decisions about management of von Willebrand Disease (VWD), a common inherited bleeding disorder.

In this symposium preceding the ASH Annual Meeting on 12/4/20, the overall objective is to inform participants about emerging diagnostic and therapeutic aspects of bleeding and thrombotic disorders. The speakers will illustrate how costly and unnecessary biochemical, phenotypic, and genetic testing can be avoided in the participants’ practices while enhancing highest quality medical care.