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.
Treatment of pyridoxamine 5'-phosphate oxidase deficiency
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Review of designation
Documents related to this orphan designation evaluation
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.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: