On 14 September 2007, orphan designation (EU/3/07/470) was granted by the European Commission to Cytonet GmbH & Co. KG, Germany, for human heterologous liver cells (for infusion) for the treatment of ornithine-transcarbamylase deficiency
The sponsorship was transferred to Promethera Biosciences, Belgium, in June 2016.
Human heterologous liver cells
Treatment of ornithine-transcarbamylase deficiency
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Documents related to this orphan designation evaluation
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.
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: