On 17 July 2013, orphan designation (EU/3/13/1163) was granted by the European Commission to Promethera Biosciences, Belgium, for heterologous human adult liver-derived progenitor cells for the treatment of argininosuccinic aciduria.
Heterologous human adult liver-derived progenitor cells
Treatment of argininosuccinic aciduria
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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.