Persistent CAR-T cells in the intestine and widespread immune disruption seen as possible drivers of a rare but serious complication Researchers at the Icahn School of Medicine at Mount Sinai have identified biological mechanisms that may contribute to CAR-T cell-associated enterocolitis, a serious inflammatory condition of the intestine that can occur after a type of
A Charité study shows that the function of defective mucosal channels nearly returns to normal Cystic fibrosis is an incurable genetic disorder. Since 2020, there has been a treatment that addresses the underlying cause, known as triple therapy, which is now also approved for children as young as two years old. In their study, researchers
The publication consolidates more than two decades of research and clinical development and confirms the long-term clinical benefit of an autologous gene therapy for Wiskott-Aldrich syndrome, a rare genetic disorder affecting the immune system and platelets. A single infusion of genetically corrected autologous hematopoietic stem cells can provide durable clinical benefit for patients with Wiskott-Aldrich
Even brief interruption tied to change in risk of heart attack, stroke and death WashU Medicine researchers found that stopping GLP-1 drugs such as semaglutide and tirzepatide — even temporarily — elevates the risk of heart attack, stroke and death compared to staying on the medication continuously. Following a rapid increase in popularity of GLP-1
A new study led by Stanford Medicine found the brain is two separate organs adjacent to one another. The finding could aid research into devastating neurological diseases. For centuries, scientists have thought of the brain as a single, unified organ. But new research led by Stanford Medicine reveals that what we call the brain is
Innovative gene therapy offers a new option for children with a rare, progressive neurodevelopmental disease The U.S. Food and Drug Administration approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A., MPS IIIA is a rare inherited disease that progressively damages the
New guidelines aim to address gaps that leave millions without timely answers or care Iron deficiency is one of the most common health conditions in the world, but many people live with symptoms for months or years before receiving a diagnosis. To address this critical unmet need, the American Society of Hematology (ASH) issued new clinical
A study by DZNE finds that healthy eating is associated with slower biological aging—and that there is more than one way to eat healthily. The researchers examined ten dietary patterns considered healthy, including the Mediterranean, Nordic, and a plant-based diet, as well as the DASH diet, which is designed to help lower blood pressure. All
A single-cell study in Science reveals major shifts in immune cells, genome organization and gene regulation in the aging human hippocampus. These findings offer critical insights into brain aging with major implications for age-related dementias. Researchers have discovered that the human brain undergoes widespread age-related changes in genome regulation beginning in midlife that may help explain why
PANXEON, which combines multiple biomarkers, also identified a precancerous condition of the pancreas, potentially opening a path to intervention before invasive cancer develops One reason pancreatic cancer remains so deadly is because it’s usually discovered late, after it has spread. Now, a major new study shows that a blood test developed by researchers led by
