On 31 July 2018, orphan designation (EU/3/18/2051) was granted by the European Commission to Wave life Sciences Ireland Limited, Ireland, for synthetic antisense oligonucleotide directed against human dystrophin pre-mRNA (also known as WVE 210201) for the treatment of Duchenne muscular dystrophy.
Synthetic antisense oligonucleotide directed against human dystrophin pre-mRNA
|Disease / condition||
Treatment of Duchenne muscular dystrophy
|Date of decision||
|Orphan decision number||
Review of designation
Sponsor's contact details
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;