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6064 results for "neurological diseases"
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.
Families facing Alexander disease, a rare and devastating neurological disorder, now have hope after the FDA approved the first-ever treatment for the condition. The new drug showed remarkable results in helping both children and adults walk better and maintain motor skills.
A one-time brain treatment that could slow Huntington's disease progression by 75% is now under review by US and UK regulators. If approved, it would be the first therapy to directly silence the gene driving this devastating neurological disorder.
Researchers identified a specific brain network driving Parkinson's disease and showed that targeting it with non-invasive treatment more than doubled symptom improvement. The discovery could transform how doctors diagnose and treat the disease affecting over 10 million people worldwide.
A breakthrough drug called AMX0035 is giving hope to 35,000 Americans living with ALS by significantly slowing the disease's progression. Research advances at Columbia's ALS Center are helping patients live longer with better quality of life. ##
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.
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 Yale scientist is discovering why some patients with myasthenia gravis don't improve with standard treatments, opening doors to personalized therapies. Her groundbreaking work could help doctors match the right medicine to each patient's unique disease pattern.
A groundbreaking cell therapy has successfully treated stiff person syndrome, a rare neurological disorder that causes severe muscle stiffness and mobility problems. If approved by the FDA this summer, it would become the first treatment for the condition and the first personalized CAR-T therapy for any autoimmune disease.
A Tennessee family is turning their son's rare disease diagnosis into a community rally for hope and research funding. The Walk for a Cure event next Saturday will support gene therapy research for PKAN, a terminal neurological condition affecting only one to three people per million worldwide.
A biotech company is preparing to test a revolutionary brain sensor that uses lab-grown neurons instead of metal electrodes, offering hope for treating conditions from stroke recovery to Parkinson's disease. Unlike traditional implants that damage brain tissue, this gentler approach could help millions suffering from neurological conditions.
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