Metabolic system
date: 24/10/2024
New medicines recommended for approval
- Wainzua (eplontersen) orphan
Treatment of hereditary transthyretin-mediated amyloidosis, a disease in which abnormal proteins called amyloids build up in tissues around the body including around the nerves
New information on approved medicines
- Cerdelga (eliglustat) - extension of indication - orphan
Treatment of Gaucher disease