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February 7, 2022: ProQR and the Usher Syndrome Coalition hosted a webinar about the newly announced clinical trials, “Sirius” and “Celeste,” for individuals with Usher syndrome type 2A (USH2A).
Sirius and Celeste are two separate Phase 2/3 studies of QR-421a, an experimental RNA therapy. Both studies will evaluate whether multiple doses of QR-421a are effective at stopping vision loss and whether they are safe and well-tolerated. The treatment is administered via intravitreal injections, or shots, directly into the eye. Sirius is for people with advanced vision loss; Celeste is for people with early to moderate vision loss. They will be recruiting approximately 200 participants, ages 12 and older, worldwide for these studies. You can find additional details in this ProQR news article
What this means to the USH community:
At the Usher Syndrome Coalition, we know that research cannot succeed without the participation of those living with Usher syndrome. Now, more than ever, is the time to be registered in the USH Trust. The Coalition will keep you informed of updates about these exciting studies.
1) Make sure that you have the most updated information in your registry record. Log in to make sure we have your preferred email address, your birthdate, and your type/subtype, if known.
2) If you have friends, family, or colleagues with Usher syndrome, please make sure they are registered in our confidential USH Trust Registry. Registration is simple, with about 10 required questions.
You do not need to have proof of a genetic diagnosis in order to register.
3) Genetic testing is the only way to know if you have Usher syndrome, and to discover the specific gene causing your subtype. If you have not yet had genetic testing, now is a good time to get it. When you get the results of your test, make sure you update your information in our USH Trust registry. Check out our website for more information on genetic testing.