September 9, 2024 by Tanesha R. Johnson, University of Kentucky Credit: Pixabay/CC0 Public DomainChildren who were diagnosed with a chronic condition at an early age face challenges that go beyond their physical health. Diseases like sickle cell have an especially stressful impact on not just the patient, but their families and caregivers as well. Sickle...
Tag: <span>Sickle Cell Disease</span>
Analysis shows gene therapy beneficial and likely cost-effective for patients with sickle cell disease
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...
Revolutionary gene therapy may offer new life for patients battling chronic sickle cell disease
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....
FDA expected to approve first CRISPR gene-editing treatment, bringing hope to thousands with sickle cell disease
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...
FDA advisors say new gene therapy for sickle cell disease is safe
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...
Sickle cell disease is 11 times more deadly than previously recorded, suggests study
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...
Portable tool to diagnose and monitor sickle cell disease receives U.S. patent
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...
New pain medications are still widely inaccessible to individuals living with sickle cell disease
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...
Bone marrow transplant may halt brain blood vessel disease in adults with sickle cell disease
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...
‘Spleen-on-a-chip’ yields insight into sickle cell disease
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...