by Justin Jackson , Medical Xpress Credit: Pixabay/CC0 Public Domain Research led by Christian Medical College in Vellore, India, has demonstrated the successful use of lentiviral vectors to deliver gene therapy for patients with severe hemophilia A. The study presents a potential alternative to adeno-associated virus (AAV)-mediated gene therapy, addressing the exclusion of patients with...
Tag: <span>Hemophilia A</span>
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Novel gene therapy for hemophilia A leads to sustained expression of clotting factor and reduced bleeding events
by Children’s Hospital of Philadelphia Lindsey A. George, MD, lead study author and attending hematologist at Children’s Hospital of Philadelphia. Credit: Children’s Hospital of Philadelphia A novel gene therapy for hemophilia A led to sustained expression of the clotting factor those patients lack, resulting in a reduction—or in some cases complete elimination—of painful and potentially life-threatening...
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The return of Vioxx: Can a drug once deemed deadly be relaunched to treat rare disease?
By DAMIAN GARDE @damiangarde OCTOBER 9, 2019 Since its recall in 2004, the pain drug Vioxx has been a symbol of pharmaceutical danger, starring in countless daytime legal advertisements explaining how you, or perhaps a loved one, might be entitled to millions in settlement dollars. But one company believes the infamous drug deserves a second...
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Hemophilia three times more prevalent than thought
by Veronica Mcguire, McMaster University More than 1,125,000 men around the world have the inherited bleeding disorder of hemophilia, and 418,000 of those have a severe version of the mostly undiagnosed disease, says a new study led by McMaster University researchers. This is three times what was previously known. Only 400,000 people globally were estimated to have the disorder which is caused...
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ASH: High-dose gene transfer beneficial in severe hemophilia A
(HealthDay)—For men with severe hemophilia A, high-dose factor VIII gene transfer is associated with sustained normalization of factor VIII activity levels, according to a study published online Dec. 9 in the New England Journal of Medicine to coincide with the annual meeting of the American Society of Hematology, held from Dec. 9 to 12 in Atlanta. Savita...