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6740 results for "genetic diseases"
Scientists matched vitamins to genetic diseases and discovered that vitamin B3 dramatically extends survival in mice with NAXD deficiency, a disorder that usually kills children within months. This genetics-first approach could unlock safe, affordable treatments for dozens of rare genetic diseases.
Over 100 scientists used Google DeepMind's new AI tool to hunt for diagnoses for 29 families living with mysterious genetic conditions. The technology can decode the 98% of human DNA that doesn't make proteins, opening new hope for 350 million people with undiagnosed rare diseases.
Two Philadelphia research teams just received millions in federal funding to develop one-dose gene therapies that could cure rare genetic diseases within five years. The breakthrough approach uses AI and new delivery methods to create treatments that work for multiple patients instead of requiring custom therapy for each person.
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.
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 40 years, treating a rare genetic lung disease meant weekly IV infusions at a clinic. Now a pill that blocks 90% of the damaging enzyme just secured a nearly half-billion dollar deal to reach American patients.
Scientists studying over one million people discovered a rare genetic mutation that slashes the risk of heart disease and diabetes by 60 percent. The discovery could lead to new treatments that protect everyone from deadly metabolic diseases.
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.
Two researchers independently discovered that ALS and frontotemporal dementia can share the same genetic cause, transforming how doctors treat these devastating conditions. Their breakthrough won them a 2026 Breakthrough Prize in Life Sciences.
A new AI algorithm called EvORanker is ending the decade-long "diagnostic odyssey" for families with rare diseases, identifying the correct disease-causing gene in 70% of test cases. The tool looks across evolution to spot hidden genetic patterns that traditional medicine misses.
A newly approved ALS treatment can now target specific genetic causes of the disease, offering hope to patients with Lou Gehrig's disease. The FDA fast-tracked the therapy, marking a significant breakthrough in personalized medicine for neurological conditions.
A new AI-powered company is helping families with ultra-rare diseases develop personalized treatments that big pharma overlooks. Founded by a rare disease patient, Nome has already guided more than 10 genetic medicine programs from diagnosis to clinical trials.
Australia's Kangaroo Island hosts the world's largest chlamydia-free koala population, but a century of isolation has left them dangerously inbred. Now scientists are using genetics to save these healthy koalas and help repopulate the struggling mainland.
Scientists are developing better treatments for idiopathic pulmonary fibrosis, a deadly lung disease that kills most patients within five years. Understanding the genetic and environmental causes is finally leading to hope for the 3 million people affected worldwide.
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.
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 a rare genetic mutation that directly causes fatty liver disease, potentially affecting millions worldwide. The breakthrough could lead to new targeted treatments for a condition that strikes one in three adults.
Scientists at Penn received $10 million to develop the first treatment for RVCL, a deadly genetic disease affecting fewer than 50 families worldwide. The new drug could prevent symptoms in people like Lindsay Ward, who's watching her mother lose her vision and mobility to the same disease she inherited.
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.
Scientists can now predict how tiny DNA changes cause diseases like diabetes and cancer, thanks to a breakthrough AI tool that reads 98% of our genetic code that was previously mysterious. The model has already helped 3,000 researchers accelerate their hunt for new treatments.
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