On 22 February 2018, orphan designation (EU/3/18/1975) was granted by the European Commission to Nightstar Therapeutics plc, the United Kingdom, for adenovirus-associated viral vector serotype 8 containing the human RPGR gene for the treatment of retinitis pigmentosa.
The sponsor’s address was updated in September 2018.
Adenovirus-associated viral vector serotype 8 containing the human RPGR 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:
- European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
- Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;