WebOn December 11, 2024, Ionis Pharmaceuticals announced the results of a small clinical trial to test the safety of a new HD drug called IONIS-HTTRx. It is the very first … Web21 uur geleden · Hope for haploinsufficiency diseases. Genetic conditions like Dravet syndrome, which causes severe childhood epilepsy, are hard to tackle with traditional gene therapy. New approaches in the works include using antisense therapy to boost mRNA splicing. The seizures started when Samantha Gundel was just four months old.
Antisense Oligonucleotides Enter Clinical Trials – HD Insights
Web7 mei 2024 · The therapy, designed by Ionis Pharmaceuticals, was the first treatment in clinical development that sought to target the underlying cause of Huntington’s disease. … Web8 mrt. 2024 · Ionis Pharmaceuticals ‘ experimental therapy IONIS-HTTRx significantly reduced levels of the protein responsible for Huntington’s disease (HD) in early stage … increase cps
IONIS-HTT Rx (RG6042) Top-Line Data Demonstrate Significant …
Web17 sep. 2024 · It’s a time of huge developments in the field of huntingtin-lowering drugs, which aim to reduce production of the huntingtin protein that causes Huntington’s disease. At the European Huntington’s Disease Network (EHDN) meeting this past week, Roche announced firm details of the first ever pivotal study of a huntingtin lowering drug – … Web22 mrt. 2024 · About tominersen and the clinical trials Tominersen, previously IONIS-HTTRx or RG6042, is an investigational antisense therapy designed to reduce the … Web23 mrt. 2024 · Huntington's disease is an unmet need with huge potential if a company can find a drug to treat the genetic disease. But Ionis has a well-stocked pipeline of drugs, including five other drug... increase cpp tax