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4654 results for "inherited disease"
Mayo Clinic scientists corrected a genetic mutation causing the most common inherited kidney disease with one injection. The treatment extended survival and stopped organ damage in mice, offering hope for 12 million people worldwide.
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 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.
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.
Scientists have developed a one-time CRISPR treatment that fixes the genetic mutation causing the most common inherited kidney disease. Early tests show it could replace lifelong medication for 12 million people worldwide.
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.
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.
A revolutionary gene therapy called Luxturna has restored vision to hundreds of people born legally blind from a rare inherited disease. One young patient saw snowfall for the first time after receiving the treatment that proved inherited diseases can be reversed.
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.
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.
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.
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.
After eliminating trachoma and other neglected tropical diseases, Malawi is leading African nations to make disease prevention a human rights issue at the UN. Twenty-five African countries have now eliminated at least one neglected tropical disease, proving progress is possible.
NYU is solving diseases faster by throwing out the old playbook: instead of keeping scientists in separate buildings, they're mixing engineers, doctors, and AI experts into teams focused on beating specific diseases. Early wins include a startup detecting airborne pathogens and "inverse vaccines" that could cure celiac disease and allergies.
Scientists at the University of British Columbia have created the first topical gene therapy that corrects faulty DNA directly in human skin, offering hope for patients with painful genetic skin disorders who currently have no cure. The breakthrough treatment could eventually help millions with conditions ranging from rare inherited diseases to common eczema.
A University of Kentucky scientist is turning Lyme disease bacteria against itself in a promising new treatment approach. The breakthrough comes as tick-borne illnesses have jumped 128% nationwide since 2020.
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