RI USH SIG – Spring Newsletter

Retina International Usher Syndrome Special Interest Group Spring Newsletter – Update on events & news relevant during the COVID-19 pandemic RI USH SIG Dominique Sturz (AT), Melissa Chaikof (US), Carol Brill (IRL), Dario Sorgato (IT) We hope that you are all fine and healthy despite the COVID-19 pandemic.
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QR-421a: Showing Benefits on One out of Four Usher Syndrome patients

Original article on AP News 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 events — QR-421a is the second ophthalmology program where clinical activity was predicted by translational models, further validating the platform — COVID-19 pandemic expected to impact timelines for the pipeline — ProQR anticipates its cash runway will fund operations into H2 2022 LEIDEN, Netherlands & CAMBRIDGE, Mass.,
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RI USH SIG Winter Newsletter

On December 2nd Retina International sent the RI USH SIG Winter Newsletter We are happy to send you the RI USH SIG Winter Newsletter providing you with an update of the latest and upcoming news relevant for people living with Usher Syndrome and with a resource file on USH subtypes, genetic testing and USH genes.
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ProQR Doses First Patient in Phase 1/2 STELLAR Trial of QR-421a for Usher Syndrome Type 2

Original text here LEIDEN, Netherlands and CAMBRIDGE, Mass., March 11, 2019 (GLOBE NEWSWIRE) — ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced the first patient dosed in the Phase 1/2 STELLAR clinical trial for QR-421a in patients with Usher syndrome type 2 or non-syndromic retinitis pigmentosa (RP).
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