On 15 October 2014, orphan designation (EU/3/14/1347) was granted by the European Commission to Great Ormond Street Hospital Foundation Trust, United Kingdom, for pyridoxal 5'-phosphate for the treatment of pyridoxamine 5'-phosphate oxidase deficiency.
|Disease / condition||
Treatment of pyridoxamine 5'-phosphate oxidase deficiency
|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.