… 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 …
… 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 …
QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse eventsQR-421a is the second ophthalmology program where clinical activity was predicted by tra
ProQR Therapeutics today announced positive results from a planned analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations.