Posted by Michael Wonder on 30 Jan 2020
ProQR receives rare paediatric disease designation from FDA for QR-421a
30 January 2020 - Designation is for the treatment of patients with retinitis pigmentosa caused by mutations in exon 13 of the USH2A gene.
ProQR Therapeutics today announced that it received Rare Pediatric Disease (RPD) designation from the United States FDA for QR-421a for the treatment of patients with retinitis pigmentosa caused by mutations in exon 13 of the USH2A gene.
QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene.
