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Usher syndrome candidate receives orphan drug designation
The FDA and the European Medicines Agency have granted orphan drug designation to QRX-421, an investigational drug for Usher syndrome, according to a press release from ProQR Therapeutics.The investigational RNA-based oligonucleotide addresses the mutations in exon 13 of the USH2A gene, which can cause loss of functional USH2A protein, causing Usher syndrome, the release said. The drug excludes exon 13 from the mRNA and produces functional USH2A protein.