On 23 August 2017, orphan designation (EU/3/17/1899) was granted by the European Commission to ProQR Therapeutics IV BV, the Netherlands, for antisense oligonucleotide targeting exon 13 in the USH2A gene (also known as QR-421) for the treatment of retinitis pigmentosa.
Antisense oligonucleotide targeting exon 13 in the USH2A gene
|Disease / condition||
Treatment of retinitis pigmentosa
|Date of first decision||
|EU designation number||
Review of designation
Sponsor's contact details
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.