ProQR’s Usher syndrome treatment gets fast track designation

The FDA has granted a fast track designation for QR-421a, an investigational treatment for vision loss associated with Usher syndrome type 2 and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene, ProQR announced in a press release.
“We are very pleased with the fast track designation the FDA granted us for QR-421a. Patients with Usher syndrome, the leading cause of combined deafness and blindness, currently have no available therapies for their vision loss and this designation emphasizes the high unmet need in this disease,” ProQR (Read more...)

Full Story →