Usher syndrome is the most common genetic cause of combined deafness and blindness. More than 400,000 people are affected by this disorder worldwide. There is currently no cure for Usher syndrome.
The Usher Syndrome Coalition is working to raise awareness and accelerate research, while providing information and support to impacted individuals and families. We strive to be the most comprehensive resource for the Usher syndrome community, bridging the gap between researchers and families. Learn more and get involved.
ProQR Therapeutics announced that the FDA has cleared the Investigational New Drug (IND) application for QR-421a. QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of the vision loss associated with Usher syndrome type 2 and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene. ProQR plans to start enrolling patients in a Phase 1/2 trial named STELLAR in the coming months with preliminary data expected in mid-2019.
We are pleased to share with you the video, transcripts, slides and summaries of the 2018 International Symposium on Usher Syndrome in Mainz, Germany.
The Usher Syndrome Coalition's one-day conference, the USH Connections Conference, is the largest annual gathering of our global Usher syndrome community. Join us for this incredible opportunity to learn the latest on developing treatments from leading USH researchers while connecting with hundreds of affected individuals, their families, and professionals serving the deafblind community.
We invite you to join the Usher Syndrome Coalition in celebration of our 4th global Usher Syndrome Awareness Day, Saturday, September 15, 2018.
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