Cambridge, UK, 2 October 2024 – LoQus23 Therapeutics Ltd (“LoQus23”), a private biotechnology company investigating small molecule drugs that could stop DNA instability and slow neurodegeneration in Huntington’s Disease, myotonic dystrophy type 1 and similar triplet repeat expansion diseases, today announces Читать дальше...
PTC518 is an oral medication that reduces the production of the mutated Huntingtin protein that causes disease progression. Programs with Fast Track designation can benefit from early interactions with the FDA, and may be eligible for priority review and accelerated approval. This follows the release of Читать дальше...
Prilenia, a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, has filed a European Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for Читать дальше...
I belong to the third generation of a family affected by Huntington’s Disease, but it wasn’t until 2024, thanks to the tireless efforts of my second father, F. Cook, that we were able to confirm how the disease entered the Читать дальше...
Abu Dhabi, UAE; June 19, 2024: IROS, an Abu Dhabi-based contract research organization (part of the M42 group), has partnered with Prilenia Therapeutics, a clinical-stage biotech company, and the International Huntington’s Disease Association (IHA) to plan the first-ever clinical trial Читать дальше...
“We are very pleased with these new data demonstrating a statistically significant, dose-dependent slowing of the progression of Huntington’s disease and lowering of NfL in the CSF at 24 months,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “We believe Читать дальше...
Сегодня компания Wave Life Sciences объявила о положительных результатах SELECT-HD, плацебо-контролируемого исследования фазы 1b/2a, в котором оценивалась исследуемая терапия WVE-003. Эти результаты показывают, что WVE-003 избирательно снижает уровень токсичного мутантного белка хантингтина (mHTT) и сохраняет здоровый белок хантингтина дикого типа (wtHTT) у людей. Читать дальше...
На 12-й месяц уровень мутантного Хантингтина (mHTT) в крови снизился на 22% и 43% соответственно для доз 5 мг и 10 мг. Аналогичный результат наблюдался и в спинномозговой жидкости, где уровень mHTT снизился на 21% и 43%, соответственно, при приеме 5 мг. Читать дальше...
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