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8124 results for "childhood disease"
Children with a rare genetic liver disorder that often leads to transplant now have their first treatment to slow disease progression. After 20 years of research, Japanese doctors turned an existing drug into a life-changing option for young patients with PFIC.
Scientists at Brown University engineered microscopic worms to help find treatments for a devastating childhood paralysis condition that currently has no cure. The breakthrough could let researchers screen thousands of potential drugs quickly and affordably.
Scientists have mapped how a tiny cellular machine malfunctions in rare childhood brain disorders, offering the first clear path toward future treatments. The discovery could help diagnose dozens of neurological conditions that currently leave families without answers.
Brown University scientists created a breakthrough worm model that could help find treatments for alternating hemiplegia of childhood, a rare disease causing paralysis in kids. The discovery offers hope to families who've waited years for effective therapies.
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.
The Jack McGovern Coats' Foundation is launching a groundbreaking effort to create the first validated animal model for Coats' Disease, a rare condition that can cause childhood blindness. This research tool could accelerate treatment development for thousands of children worldwide.
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.
Scientists at Brunel University and UCL have developed a stem cell treatment that could prevent Friedreich's ataxia before symptoms appear. In mice, the therapy restored near-normal protein levels and improved movement, offering hope for children facing this devastating condition.
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.
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.
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