Last reviewed · How we verify
Lateonset Pompe disease
Approved treatments
- Zavesca · AstraZeneca
Zavesca works by inhibiting the enzyme glucosylceramide synthase, which is involved in the production of a toxic lipid that accumulates in cells of patients with certain lysosomal storage disorders.
Competitive intelligence
For the full treatment-landscape CI brief — marketed leaders side-by-side, pipeline by phase, recent regulatory actions, sponsor landscape:
- Lateonset Pompe disease patent landscape — cliff calendar, originator estates, attackable patents
- Lateonset Pompe disease treatment landscape brief
- Lateonset Pompe disease treatment updates RSS
Frequently asked questions about Lateonset Pompe disease
What drugs treat Lateonset Pompe disease?
Tracked treatments include Zavesca.
What is the treatment for Lateonset Pompe disease?
Standard treatment for Lateonset Pompe disease includes Zavesca. See the treatment landscape page for first-line, second-line and salvage options.
Related
- Treatment landscape: Lateonset Pompe disease patent cliff · Lateonset Pompe disease CI report
- Treatment: Zavesca · AstraZeneca
- All diseases: Browse the disease index · Disease landscape hub
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing