… the creation of transformative RNA therapies for severe genetic rare diseases, today announced that the Company will … pigmentosa. They will discuss the role of genetictesting and outcome measures related to visual field and …
… the creation of transformative RNA therapies for severe genetic rare diseases, today reported its financial results … program in the United States providing no-cost genetictesting and genetic counseling for individuals with a …
… the creation of transformative RNA medicines for severe genetic rare diseases, today reported its financial results … We advanced two additional programs to Phase 1/2 clinical testing – QR-421a for Usher syndrome and non-syndromic RP, …
Phase 2/3 pivotal Illuminate trial of sepofarsen for LCA10 enrollment complete with top-line data anticipated in H1 2022; Brighten pediatric trial of sepofarsen to begin in 2021; additional data from Phase 1/2 InSight extension study to be reported in