Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in June 2018 on request of the Sponsor.
On 16 October 2015, orphan designation (EU/3/12/972) was granted by the European Commission to Ultragenyx UK Limited, United Kingdom, for sialic acid (also known as aceneuramic acid) for treatment of GNE myopathy.
The sponsorship was transferred to Ultragenyx Germany GmbH, Germany, in January 2017.
Sialic acid (also known as aceneuramic acid)
|Disease / condition||
Treatment of GNE myopathy
|Date of first decision||
|EU designation number||
Review of designation
Sponsor's contact details
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;
- European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.