On 11 January 2019, orphan designation (EU/3/18/2125) was granted by the European Commission to Isabelle Ramirez, Germany, for benserazide hydrochloride for treatment of sickle cell disease.
The treatment of sickle cell disease
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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.
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: