On 17 January 2018, orphan designation (EU/3/17/1965) was granted by the European Commission to Duchenne UK, United Kingdom, for metformin and L-citrulline for the treatment of Duchenne muscular dystrophy.
The sponsorship was transferred to Quality Regulatory Clinical Ireland Limited, Ireland, in July 2019.
Treatment of Duchenne muscular dystrophy
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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: