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 Prečítajte si viac...
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 Prečítajte si viac...
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 Prečítajte si viac...
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 Prečítajte si viac...
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 Prečítajte si viac...
“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 Prečítajte si viac...
Spoločnosť Wave Life Sciences dnes oznámila pozitívne výsledky štúdie SELECT-HD, našej placebom kontrolovanej štúdie fázy 1b/2a, ktorá hodnotí skúmanú liečbu WVE-003. Tieto výsledky dokazujú, že WVE-003 selektívne znižuje toxický mutantný proteín huntingtinu (mHTT) a zachováva zdravý proteín huntingtinu divokého typu (wtHTT) u jedincov Prečítajte si viac...
V 12. mesiaci sa mutantný huntingtín (mHTT) v krvi znížil o 22% a 43% pri dávkach 5 mg a 10 mg. Podobný výsledok bol pozorovaný v mozgovomiechovom moku, kde sa mHTT znížil o 21% a 43% pre 5 mg, resp. Prečítajte si viac...
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