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28 results for "genetic disease"
Scientists who created gene therapies that cure inherited blindness and blood disorders just won six $3 million prizes. Their discoveries are transforming devastating diseases from hopeless diagnoses into treatable conditions.
People with a rare genetic disorder that causes dangerous fat buildup in their blood now have a breakthrough treatment that cuts triglyceride levels by nearly a third. The UK just approved olezarsen for adults with familial chylomicronemia syndrome, a condition that can trigger life-threatening pancreas inflammation.
A Colorado scientist's decade of detective work just gave hope to families battling Blau syndrome, a genetic disease affecting fewer than 1,000 Americans. Two patients are already seeing remarkable results.
At 32, Yentli Soto Albrecht has a 95% chance of developing the same fatal disease that killed her father. She's managing 11 research projects to find a cure in time.
A medical student who inherited the genetic mutation that killed her father is now leading eleven research projects to cure ALS before it affects her. Yentli Soto Albrecht has a 95% chance of developing the fatal disease, but she's turning her fate into fuel for breakthrough research.
University of Virginia researchers used gene editing to fix the DNA mutation causing a deadly form of epilepsy, potentially opening the door to cures for inherited brain disorders. The technique eliminated seizures in lab mice and improved their movement and cognitive function.
Jeff Vierstra lost his mother and two sisters to a deadly genetic disease, but an experimental treatment may be preventing him from developing ALS for the first time in his family's history. After three years of spinal infusions targeting his mutated gene, the 41-year-old shows no signs of the disease.
A bacterial defense system discovered in 2012 has transformed into life-saving treatments for cancer and rare diseases. Scientists refined CRISPR gene editing from crude molecular scissors into precision medicine that's curing patients today.
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.
The FDA just approved Denali Therapeutics' new medicine for Hunter syndrome, bringing fresh hope to families affected by this rare genetic disease. The approval comes as a welcome surprise after the agency recently toughened its standards for rare disease treatments.
Children with Hunter syndrome just got their first new treatment option in nearly two decades. The FDA approved AVLAYAH, a breakthrough therapy from Denali Therapeutics that offers fresh hope to families facing this rare genetic disease.
Researchers discovered how to disguise healthy mitochondria so they can slip into diseased cells undetected, extending the lives of mice with a fatal genetic disorder. The breakthrough could one day help children born with rare mitochondrial diseases.
The FDA just unveiled a breakthrough framework that could bring life-saving gene therapies to thousands of children with rare diseases in a fraction of the time. For families who've watched their kids suffer while treatments stall, this changes everything.
Researchers have identified a tiny protein region that keeps hearts beating steadily throughout our lives. The breakthrough could lead to treatments for devastating inherited heart diseases that affect thousands of families.
Ty Sperle, 18, became the first person in the world cured of chronic granulomatous disease through groundbreaking gene editing treatment. His success opens the door for curing countless other rare genetic diseases affecting children everywhere.
Scientists have created a precise gene-editing tool that could offer permanent treatment for cystic fibrosis patients, potentially replacing daily medications that cost hundreds of thousands of dollars per year. The breakthrough could transform care for people living with this and other genetic diseases.
A 9½-month-old baby became the first person ever to receive a custom gene-editing treatment designed specifically for his unique mutation. The breakthrough offers hope for thousands of rare genetic diseases that were once considered unreachable.
Adults with a rare genetic disease called cystinosis no longer need dozens of pills each day after receiving a groundbreaking one-time gene therapy. Five years later, most patients show stable or improved health across multiple organs.
Scientists have developed a new gene editing approach that spreads healing changes from cell to cell, dramatically increasing its reach in the body. This breakthrough could transform treatment for countless genetic diseases that were previously too difficult to address.
Scientists are developing a breakthrough gene editing approach that spreads between cells like neighbors sharing flyers. This could treat far more diseases by reaching dramatically more cells in the body.
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