On 22 February 2018, orphan designation (EU/3/18/1973) was granted by the European Commission to ProQR Therapeutics IV BV, the Netherlands, for 2'-O-(2-methoxyethyl)-modified antisense oligonucleotide targeting exon 13 in the USH2A gene (also known as QR 421a) for the treatment of retinitis pigmentosa.
2'-O-(2-Methoxyethyl)-modified antisense oligonucleotide targeting exon 13 in the USH2A gene
|Disease / condition||
Treatment of retinitis pigmentosa
|Date of decision||
|Orphan decision number||
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.