… cystic fibrosis, Leber's congenital amaurosis 10 and dystrophic epidermolysis bullosa, today announced details … Brompton Hospital, and immediate past-president of the European Cystic Fibrosis Society, will give an oral … QR-010 is designed to be self-administered via an optimized eFlow ® Nebulizer (PARI Pharma GmbH). eFlow ® is a …
… ProQR has granted an exclusive license to Amylon to develop therapeutics for beta amyloid related disorders and … to future milestones and royalties from the products developed. Amylon will initially focus on developing a RNA-based therapy for a rare genetic disease …
… in clinical development with the planned Phase 1/2 open-label trial (PQ-110-001) that will assess the safety, … 6 adults (≥ 18 years) that have LCA 10 due to one or two copies of the p.Cys998X mutation. During the trial, patients … expertise in genetic retinal disease in the US and Europe. The primary endpoints will be safety and …
… are no therapies commercially available The Phase 1/2 open-label trial will assess the safety, tolerability, … 6 adults (≥ 18 years) that have LCA 10 due to one or two copies of the p.Cys998X mutation. During the trial, patients … expertise in genetic retinal disease in the US and Europe. The primary endpoints will be safety and …
… PQ-110-001 Phase 1/2 Trial PQ-110-001 is a first-in-human open-label trial that enrolled 5 children (age 6 - 17 years) … and 6 adults (≥ 18 years) who have LCA10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. Patients … of visual function and retinal structure through ophthalmic endpoints, such as visual acuity (BCVA), mobility …
LEIDEN, Netherlands & CAMBRIDGE, Mass., May 12, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced that it will p
… for AX-0810 AX-0810 is an editing oligonucleotide (EON) development program targeting the SLC10A1 RNA using the … biomarker bile acids 72 hours after treatment. Further optimizations for EONs targeting NTCP have enabled … models and showed translatability across models. ProQR is developing its AX-0810 program targeting NTCP for Cholestatic …
… le Professeur Samuel G. Jacobson, M.D., Ph.D., qui est l’ophtalmologiste qui s’occupe de quatre des patients inclus … » devrait être lancée au cours du premier semestre 2019. À propos de l’amaurose congénitale de Leber 10 L’amaurose … occidental souffrent de LCA10 à cause de cette mutation. À propos du QR-110 Le QR-110 est un premier oligonucléotide …
… Study of Homozygous F508del Cystic Fibrosis Patients Topline data to be presented today at NACFC LEIDEN, the … QR-010 is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). eFlow® is a … orphan drug designation in the United States and the European Union and fast-track status by the FDA. The QR-010 …