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12 results for "genetic disorders"
Five people with a life-threatening blood disorder no longer need regular transfusions after receiving a groundbreaking gene therapy in China. The treatment could bring hope to 350 million carriers of thalassaemia worldwide.
Children with rare genetic growth disorders grew twice as fast after starting a groundbreaking treatment, offering hope where few options existed before. The therapy worked especially well for kids whose condition directly matched the drug's target.
Researchers discovered how to lower dangerous cholesterol without relying on damaged receptors, offering hope to 1 in 200 adults with a common genetic disorder. The breakthrough uses human liver cells to test compounds that stop cholesterol particles from forming in the first place.
Twenty-seven out of 28 patients with severe sickle cell disease are now living pain-free after a groundbreaking gene editing treatment. The one-time therapy is offering new hope to people living with a genetic disorder that typically shortens life expectancy to the mid-40s.
Around 20 people in England with a debilitating genetic condition can now access the first treatment that targets the root cause of their disease. The NHS will provide pegzilarginase to patients with arginase-1 deficiency after breakthrough approval.
Scientists just diagnosed a rare genetic condition in a mother and daughter who lived over 12,000 years ago, proving that communities cared for vulnerable members even during the Ice Age. The discovery marks the earliest genetic diagnosis ever made in human history.
A rare disease nonprofit co-founded by Pearl Jam's Eddie Vedder is revolutionizing medical research by acting like a venture capital firm, turning every dollar into multiple cures. Their innovative funding model has already contributed to three FDA-approved treatments for a devastating skin disease that once had no hope.
Virginia Tech researchers discovered early brain changes in Leigh syndrome that could help doctors identify affected children months before symptoms appear. The breakthrough offers hope for earlier treatment of this devastating genetic disorder.
Jeffrey Randall Allen won $10 million on Amazon's Beast Games and immediately directed millions toward researching a cure for his son Lucas's rare genetic disorder. His mission is bringing unprecedented funding and attention to Creatine Transporter Deficiency, a condition that affects how the brain accesses a critical nutrient.
A decades-old sleeping sickness drug is showing promise for children with Bachmann-Bupp syndrome after doctors treated six patients with remarkable results. Now a major partnership could fast-track treatments for the 20 known cases worldwide.
The FDA just approved the first treatment for Menkes disease, a rare genetic disorder that has claimed countless young lives. Copper histidinate could help babies with this devastating condition finally have a fighting chance.
Scientists at Carnegie Mellon University have discovered a precise way to target toxic RNA that causes myotonic dystrophy type 1, the most common adult-onset muscular dystrophy affecting 1 in 2,300 people worldwide. The breakthrough could lead to treatments with fewer side effects for this currently incurable disease and other devastating genetic disorders.