EU/3/18/1973

Table of contents

About

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.

Key facts

Active substance
2'-O-(2-Methoxyethyl)-modified antisense oligonucleotide targeting exon 13 in the USH2A gene
Disease / condition
Treatment of retinitis pigmentosa
Date of decision
22/02/2018
Outcome
Positive
Orphan decision number
EU/3/18/1973

Sponsor's contact details

ProQR Therapeutics IV BV
Zernikedreef 9
2333 CK Leiden
The Netherlands
Tel. +31 88 166 7000
E-mail: info@proqr.com

Patients' organisations

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.

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