On 9 December 2020, orphan designation EU/3/20/2365 was granted by the European Commission to Sarepta Therapeutics Ireland Limited, Ireland, for adeno-associated virus serotype rh74 containing the human sarcoglycan beta gene (also known as SRP-9003) for the treatment of limb-girdle muscular dystrophy.
Adeno-associated virus serotype rh74 containing the human sarcoglycan beta gene
Treatment of limb-girdle muscular dystrophy
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
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: