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 قراءة المزيد…...
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 قراءة المزيد…...
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 قراءة المزيد…...
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 قراءة المزيد…...
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 قراءة المزيد…...
“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 قراءة المزيد…...
أعلنت اليوم شركة "ويف لايف ساينسز" عن نتائج إيجابية من تجربة "سيليكت- إتش دي"، وهي المرحلة 1 ب/2 أ من تجربتنا الخاضعة للتحكم بالعلاج الوهمي لتقييم العلاج التجريبي WVE-003. وتظهر هذه النتائج أن WVE-003 يقلل بشكل انتقائي من بروتين هانتينتين السامة الطافرة (mHTT) ويحافظ على بروتين هانتينتين السليم من النوع البري (wtHTT) للأفراد قراءة المزيد…...
في الشهر 12، انخفضت نسبة الهانتينجتين الطافرة (mHTT) في الدم بمقدار 22% و43% على التوالي لجرعات 5 ملغ و10 ملغ. ولوحظت نتيجة مماثلة في السائل الدماغي الشوكي، حيث تم خفض mHTT بمقدار 21% و43% على التوالي لجرعة 5 ملغ و10 ملغ قراءة المزيد…...
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