On 26 February 2019, orphan designation (EU/3/19/2145) was granted by the European Commission to Roche Registration GmbH, Germany, for risdiplam for the treatment of spinal muscular atrophy.
|Disease / condition||
Treatment of spinal muscular atrophy
|Date of first decision||
|EU designation number||
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.