by American College of Physicians Normal blood cells next to a sickle-blood cell, colored scanning electron microscope image. Credit: Wikipedia/Illustration from Anatomy & PhysiologyA modeling study comparing the cost-effectiveness of gene therapy versus common care for patients with sickle cell disease found that gene therapy is beneficial in this patient population and likely cost-effective if the...
by Robert McCoppin, Chicago Tribune Normal blood cells next to a sickle-blood cell, colored scanning electron microscope image. Credit: Wikipedia/Illustration from Anatomy & PhysiologyLyric Porter wanted her life back. Born with sickle cell disease, she had a fever at age 5 that scarred her lungs and required her to roll an oxygen tank to school....
By Meg Tirrell, CNN Since Johnny Lubin got the exa-cel treatment in October 2021, “he’s been like a normal child,” his mother says. “It’s life-changing for us.”Courtesy JR Lubin and Fabienne DesirCNN — Before Johnny Lubin got a potentially life-changing experimental treatment at the age of 13, he recalls, he had one main concern. “I was...
by Physician’s Briefing Staff A new gene therapy for sickle cell disease has been deemed safe by a U.S. Food and Drug Administration advisory panel, paving the way for full approval by early December 2023. The FDA had already decided that the therapy, known as exagamglogene autotemcel (exa-cel), was effective. Developed by Vertex Pharmaceuticals of...
by Institute for Health Metrics and Evaluation Credit: National Institutes of Health A new study suggests the number of deaths due to sickle cell disease is 11 times higher than what is indicated from mortality data sources alone. Sickle cell disease is not just underdiagnosed, but it also increases risk of infection and of death from...
FLORIDA ATLANTIC UNIVERSITY IMAGE: SARAH DU, PH.D., INVENTOR AND AN ASSOCIATE PROFESSOR, FAU COLLEGE OF ENGINEERING AND COMPUTER SCIENCE. CREDIT: ALEX DOLCE, FLORIDA ATLANTIC UNIVERSITY In the United States, sickle cell disease affects about 100,000 people and about 2 million Americans carry this genetic mutation. The most common and serious problems caused by sickle cell...
AMERICAN SOCIETY OF HEMATOLOGY (WASHINGTON, March 8, 2023) – Sickle cell disease (SCD), a rare chronic, progressive, life-threatening, inherited blood disorder, often affects individuals with chronic pain that can be debilitating to their quality of life. Yet less than 4% of people living with SCD who experience chronic pain episodes have prescriptions for newer FDA-approved...
by American Heart Association Credit: Pixabay/CC0 Public Domain Bone marrow transplants prevented or improved blood vessel disease in the brains of adults with sickle cell disease, according to preliminary research to be presented at the American Stroke Association’s International Stroke Conference 2023. The meeting, to be held in person in Dallas and virtually Feb. 8-10, 2023, is...
by Massachusetts Institute of Technology Credit: Pixabay/CC0 Public Domain Every day, billions of red blood cells pass through the spleen, an organ that is responsible for filtering out old or damaged blood cells. This task is made more difficult when the blood cells are misshapen, as they are in patients with sickle cell disease, which...
ST. JUDE CHILDREN’S RESEARCH HOSPITAL IMAGE: CORRESPONDING AUTHOR MITCHELL J WEISS, M.D., ST. JUDE DEPARTMENT OF HEMATOLOGY CHAIR; FIRST AUTHOR RUOPENG FENG, PH.D., ST. JUDE DEPARTMENT OF HEMATOLOGY CREDIT: COURTESY OF ST. JUDE CHILDREN’S RESEARCH HOSPITAL (MEMPHIS, Tenn. – October 12, 2022) Scientists at St. Jude Children’s Research Hospital have shown how a protein responsible...
