Stable and Durable Factor VIII Expression in Subjects Dosed with Investigational Hemophilia A Gene Therapy
More than two years after receiving Spark Therapeutics’ investigational adeno-associated virus (AAV)-mediated gene therapy (SPK-8011), five adult subjects with hemophilia A exhibited stable factor VIII (FVIII) expression, with no evidence of FVIII inhibitors or a cellular immune response.
Breakthroughs in Stem Cell Research
Optimism continues to grow about the use of stem cells in regenerative medicine highlighted by hundreds of successful clinical trials.
Exploring New Vaccine Delivery Methods
Some promising new methods for delivering vaccinations could mean a pain-free alternative, the elimination of accidental needlesticks and reduced risk of infections.
High-Titer Inhibitors Reduced in PUPs with Severe Hemophilia A Treated with Fourth Generation Recombinant Factor VIII
A study in previously untreated patients (PUPs) with severe hemophilia A found replacement therapy with a fourth-generation recombinant factor VIII (FVIII) product was associated with an incidence of high-titer inhibitors similar to the rate seen in a landmark trial evaluating plasma-derived FVIII products.
CDC Analyses Show Flu Vaccine Reduces Risk of Hospitalization in Children and Death in Adults
Two analyses by the Centers for Disease Control and Prevention (CDC) show the influenza (flu) vaccine can reduce the likelihood of hospitalization in children and death in adults.
Study Finds Safe Treatment for Children with Severe Hemophilia A with Inhibitors
Researchers have found combining immune tolerance induction (ITI) with Hemlibra (emicizumab, Roche) is a feasible and safe way of treating children with severe hemophilia A.
Flu Vaccine May Reduce Risk of Heart Attack and Stroke in Hypertensive Individuals
A new study shows cardiovascular outcomes may improve in individuals with hypertension if they receive the influenza (flu) vaccine during the flu season.
Skin Patch Shows Promise for Delivery of Influenza Vaccine
Researchers at the University of Rochester Medical Center in New York City have developed a new type of skin patch that could replace needles as a method to deliver the influenza (flu) vaccine.
Researchers Uncover How Human Immunity Evades Influenza Virus
A new study that looked at how the influenza (flu) virus escapes antibodies found the site of the escape mutation varies among most individuals’ sera.
Plasma-Derived ApoA-1
Findings suggest CSL112 can boost cholesterol efflux capacity in patients with impaired endogenous HDL function. Encouragingly, several studies have documented its ability to reduce atherosclerotic plaque volume.
