US EditionUniQure’s Gene Therapy Slows Huntington’s Disease Progression by 75%
- UniQure announced this week that its experimental gene therapy AMT-130 slowed Huntington's disease progression by 75% over 36 months in a clinical trial at University College London.
- This trial follows earlier failures of antisense therapy targeting Huntington's disease and uses a one-time gene therapy to silence the faulty huntingtin gene causing the disorder.
- The trial treated 29 early-stage patients, with 17 receiving a high dose through a complex brain surgery lasting up to 18 hours, injecting a harmless virus to deliver microRNA that blocks the toxic huntingtin protein.
- Dr. Walid Abi-Saab stated on Wednesday that AMT-130 could dramatically change how Huntington's disease is treated, while other specialists described the findings as "spectacular" and "very promising."
- UniQure plans to seek FDA approval by 2026 despite treatment complexity and cost, and researchers are planning larger and preventative trials, signaling potential hope for altering Huntington's disease and similar disorders.
209 Articles
209 Articles
Huntington's Disease Successfully Treated For First Time
Every day, modern science makes breakthroughs, tackling debilitating and fatal diseases. For the first time, Huntington’s disease has been treated, marking a huge step for people suffering from and susceptible to it. Huntington’s is a dreadfully cruel hereditary disease that slowly destroys a person’s brain until they are completely debilitated. It is a brutal end to someone’s life, and it is a devastating ordeal for families. Like many other de…
Scientists Just Announced the First Real Treatment for Huntington’s Disease
For decades, a Huntington’s disease diagnosis has been a death sentence. It shows up like a wrecking ball in midlife, disrupting motor function, cognition, and emotional stability. It grinds its victims down over 10 to 25 brutal years, with treatments only masking symptoms. However, after years of work and numerous failed trials, researchers may have finally developed a viable treatment. In clinical trial results released on September 24, a gene…
New hope for Huntington’s families as gene therapy shows remarkable results
kazoka/Shutterstock.comA company called uniQure has announced promising results from a trial of a new gene therapy for Huntington’s disease. The news has spread quickly through families affected by this condition, who have been desperately waiting for a treatment that can stop or slow down this devastating illness. Huntington’s disease is a fatal brain disorder that runs in families, caused by a faulty gene that produces a protein called hunting…
For the first time, gene therapy injected into the brain has significantly slowed the progression of the genetically inherited Huntington's disease, reports Nature.
A study raises hope for those affected by the serious hereditary disease. The technology behind it may be used to treat other diseases in the future.
Coverage Details
Bias Distribution
- 67% of the sources are Center
Factuality
To view factuality data please Upgrade to Premium
