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5484 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.
Scientists are using a safer twist on gene editing to treat diseases by flipping genetic switches instead of cutting DNA. The first trial results for a rare muscular disorder just arrived, and they're opening doors for conditions from high cholesterol to inherited diseases.
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.
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.
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.
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 one-time gene therapy targeting a devastating inherited heart condition just earned special FDA status, bringing hope to families facing early heart failure. The treatment addresses the root genetic cause rather than just managing symptoms.
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.
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.
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.
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