Find uplifting stories about heroes, innovations, and solutions
6100 results for "hereditary disease"
Scientists used advanced AI to discover why Huntington's disease begins at vastly different ages in patients, finding that genetic context matters more than previously understood. The breakthrough could lead to personalized treatments for this and other hereditary diseases.
For the first time ever, children and adults with Alexander disease have a treatment that attacks the root cause of their condition, not just the symptoms. The FDA's approval of zilganersen gives hope to families facing a disease that has always been fatal.
A groundbreaking treatment for Huntington's disease just cleared a major hurdle, bringing hope to 240,000 Americans affected by this devastating condition. The experimental therapy could become the first disease-modifying treatment for a disorder that has had none.
A doctor who survived a rare disease now uses artificial intelligence to find unexpected treatments that help patients like Kaila Mabus, who was told she might not survive. His AI system tests 75 million possible drug-disease combinations in minutes.
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.
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.
A new treatment for a rare kidney disease just passed a major test, bringing hope to 330,000 patients in the U.S. and Europe. Vertex's drug successfully reduced a key disease marker by half in late-stage trials.
University of Queensland researchers have developed a breakthrough drug that could transform Motor Neurone Disease from a terminal diagnosis into a manageable chronic condition within five years. The discovery unlocks a previously untargetable immune receptor that plays a key role in neurodegenerative diseases.
Pearl Jam's Eddie Vedder and Canadian director Matt Finlin created a documentary following the race to cure epidermolysis bullosa, a devastating skin disease affecting children worldwide. The Netflix film captures breakthrough research that could unlock treatments for thousands of rare diseases.
For the first time, a gene therapy has shown dramatic results in slowing Huntington's disease, offering hope to 8,000 UK families who've had no treatment options until now. Patients receiving the higher dose experienced 75% less disease progression over three years.
Four leading European research institutes have joined forces to speed up treatments for Alzheimer's, Parkinson's, and other brain diseases affecting millions. The CURE-ND Alliance brings together over 2,500 scientists working to transform how we understand and treat neurodegenerative diseases.
Nancy Wexler discovered her mother's illness was hereditary and she had a 50% chance of getting it. Instead of retreating, she led the groundbreaking research that found the gene responsible for Huntington's disease.
A Nashville family transformed their annual Kentucky Derby party into a powerful fundraiser after their daughter was diagnosed with an incurable nerve disease. Last year alone, they raised $75,000 for research into Charcot-Marie-Tooth disease.
A groundbreaking gene therapy called AMT-130 has slowed Huntington's disease progression by 75% in clinical trials, offering hope to families who've waited 154 years for effective treatment. The one-time treatment could preserve patients' ability to walk, speak, and connect with loved ones for years longer.
Jeff Carroll learned he carried the gene for deadly Huntington's disease. Instead of giving up, he became a neuroscientist dedicated to finding a cure for the condition killing him.
After initially calling it a failed product, the FDA reversed course to allow accelerated approval of a groundbreaking gene therapy that could slow Huntington's disease. The decision brings hope to 40,000 Americans living with the fatal disorder that currently has no cure.
The FDA has accepted a groundbreaking treatment for Alexander disease for priority review, offering hope to families battling a rare neurological condition that has no approved therapies. If approved by September, zilganersen would become the first medicine ever available for this devastating illness.
The FDA reversed course on a groundbreaking gene therapy for Huntington's disease, bringing new hope to thousands of families. The treatment from biotech company UniQure can now move forward toward U.S. approval.
Deaths from coronary artery disease have dropped more than 50% in the United States over three decades, thanks to better control of key risk factors like smoking and air pollution. Even better news: nearly 90% of remaining deaths could still be prevented.
A promising treatment for microvillus inclusion disease, a deadly childhood condition, has entered breakthrough therapy talks with the FDA. The oral medication has already shown it can reduce life-sustaining interventions in young patients.
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