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8 results for "genetic therapy"
Yale researchers created a groundbreaking database of 520 FDA-approved drugs by studying tiny zebrafish, revealing potential personalized treatments for people with specific autism-related genetic mutations. One drug candidate successfully reversed disrupted behaviors in both fish and human brain cells.
Children with a devastating form of epilepsy may soon have new hope thanks to promising trial results from an experimental treatment. Praxis Precision Medicines announced their drug elsunersen successfully reduced seizures in early testing for SCN2A-related epilepsy.
Scientists just achieved a major breakthrough in treating two rare muscle diseases that currently have no cure. Early trials show a new delivery method gets medicine directly into muscle cells without serious side effects.
Families affected by Hunter syndrome now have hope as the FDA approves Avlayah, the first treatment designed to replace a missing enzyme that causes dangerous sugar buildup in the brain. This breakthrough marks a major victory for patients with this rare genetic disease.
Researchers at UConn have created a promising new therapy that could stop and even reverse fatty liver disease, a condition affecting billions globally with few current treatment options. The innovative approach targets a master regulator in the liver that controls over 1,000 genes crucial for metabolic function.
A groundbreaking treatment could help children with Dravet syndrome not just reduce seizures, but actually improve cognitive development and daily living skills. For the first time, families facing this devastating genetic condition have hope for a disease-modifying therapy.
A groundbreaking treatment for myotonic dystrophy type 1 reduced toxic proteins by 40% and improved muscle function in patients who've never had disease-modifying options. The New England Journal of Medicine just published results that could lead to the first approved therapy for this progressive, often fatal disease.
Scientists discovered how to permanently switch off cancer-causing genes, potentially shortening treatment and reducing harsh side effects. The breakthrough targets aggressive leukemia and enters clinical trials this year.