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 Διαβάστε περισσότερα...
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 Διαβάστε περισσότερα...
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 Διαβάστε περισσότερα...
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 Διαβάστε περισσότερα...
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 Διαβάστε περισσότερα...
“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 Διαβάστε περισσότερα...
Σήμερα, η Wave Life Sciences ανακοίνωσε θετικά αποτελέσματα από την SELECT-HD, την ελεγχόμενη με εικονικό φάρμακο δοκιμή φάσης 1b/2a που αξιολογεί την ερευνητική θεραπεία WVE-003. Τα αποτελέσματα αυτά καταδεικνύουν ότι το WVE-003 μειώνει επιλεκτικά την τοξική, μεταλλαγμένη πρωτεΐνη huntingtin (mHTT) και διατηρεί την υγιή, άγριου τύπου πρωτεΐνη huntingtin (wtHTT) για τα άτομα Διαβάστε περισσότερα...
Τον μήνα 12, η μεταλλαγμένη Huntingtin (mHTT) στο αίμα μειώθηκε κατά 22% και 43% αντίστοιχα για τις δόσεις των 5mg και 10mg. Παρόμοιο αποτέλεσμα παρατηρήθηκε και στο εγκεφαλονωτιαίο υγρό, όπου η mHTT μειώθηκε κατά 21% και 43% αντίστοιχα, για 5mg Διαβάστε περισσότερα...
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