A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers prepare to meet growing ...
Dec 6, 2025
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Researchers at the Eye Genetics Research Unit at Children's Medical Research Institute (CMRI) are the first in the world to use stem cells to study one of the genetic causes of Leber Congenital Amaurosis (LCA)—a rare condition ...
Nov 20, 2025
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Alzheimer's disease (AD) is a devastating cause of memory loss and cognitive decline, for which no curative treatment is available. Among lifestyle factors, physical activity stands out as possibly one of the strongest defenders ...
Nov 18, 2025
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The United States on Friday said it would add its most serious warning label to a gene therapy treatment for a debilitating muscle-wasting disease, and also limit its approved use.
Nov 15, 2025
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Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a rare liver disorder.
Nov 14, 2025
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Rice University bioengineers have demonstrated a nonsurgical way to quiet a seizure-relevant brain circuit in an animal model. The team used low-intensity focused ultrasound to briefly open the blood-brain barrier (BBB) in ...
Nov 11, 2025
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At Mayo Clinic, cardiologists Peter Noseworthy, M.D., and John Giudicessi, M.D., Ph.D., are uncovering the earliest signs of a genetic heart condition that often strikes without warning. Their work shows that early detection, ...
Nov 11, 2025
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The malignant pediatric brain tumor medulloblastoma is driven by proteins deep within the cancer cells, for which there are often no effective drugs available. Researchers at Uppsala University have identified a new genetic ...
Nov 10, 2025
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Batten disease is a rare inherited condition that affects brain development and function. CLN3 disease is the most common type of this disease. The symptoms are life-changing. They usually begin between the ages of four and ...
Nov 6, 2025
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Around the globe, heart disease remains one of the top causes of death. Once patients begin to suffer from serious heart problems, like heart attacks and heart failure, the heart muscles become damaged and are difficult to ...
