The Usher Syndrome Coalition announces partnership with ProQR to support clinical trial enrollment for a potential therapy for USH2A mediated retinitis pigmentosa.
WESTFORD, Mass., Jan. 31, 2022 /PRNewswire/ -- For more than ten years, the Usher Syndrome Coalition has been working to build its international "USH Trust" registry in anticipation of the day when researchers would launch a final-stage clinical trial for the Usher syndrome community. That time has come. The Coalition is pleased to partner with ProQR to help recruit 200+ participants worldwide for this exciting study.
Clinical trials Sirius and Celeste aim to determine whether the investigational RNA therapy QR-421a is effective at stopping vision loss and is safe for people with retinitis pigmentosa and Usher syndrome due to mutations in exon 13 of the USH2A gene. These Phase 2/3 clinical trials have been planned after positive findings in a Phase 1/2 clinical trial named Stellar. More information on Stellar can be found here: www.proqr.com/community-stories-and-news/positive-results-achieved-in-our-ongoing-usher-syndrome-and-retinitis-pigmentosa-research.