Last reviewed · How we verify
Duchenne muscular dystrophy
Approved treatments
- Viltepso · Nippon Shinyaku
Viltepso works by binding to exon 53 of dystrophin pre-mRNA, allowing for the production of a partially functional dystrophin protein. - Exondys 51 · Sarepta
Exondys 51 binds to specific RNA sequences to prevent the skipping of exon 51 during dystrophin mRNA splicing. - Vyondys 53 · Sarepta
Vyondys 53 works by binding to specific RNA sequences to prevent the production of a faulty dystrophin protein. - Amondys 45 · Sarepta
Amondys 45 works by binding to specific RNA sequences to prevent the production of a faulty protein that causes Duchenne muscular dystrophy. - Emflaza · Ptc Therap
Emflaza works by mimicking the effects of cortisol, a natural hormone produced by the body, to reduce inflammation and suppress the immune system. - Translarna · PTC Therapeutics International Limited
Competitive intelligence
For the full treatment-landscape CI brief — marketed leaders side-by-side, pipeline by phase, recent regulatory actions, sponsor landscape:
- Duchenne muscular dystrophy patent landscape — cliff calendar, originator estates, attackable patents
- Duchenne muscular dystrophy treatment landscape brief
- Duchenne muscular dystrophy treatment updates RSS
Frequently asked questions about Duchenne muscular dystrophy
What drugs treat Duchenne muscular dystrophy?
Tracked treatments include Viltepso, Exondys 51, Vyondys 53, Amondys 45, Emflaza, Translarna.
What is the treatment for Duchenne muscular dystrophy?
Standard treatment for Duchenne muscular dystrophy includes Viltepso, Exondys 51, Vyondys 53, Amondys 45, Emflaza. See the treatment landscape page for first-line, second-line and salvage options.
Related
- Treatment landscape: Duchenne muscular dystrophy patent cliff · Duchenne muscular dystrophy CI report
- Treatment: Viltepso · Nippon Shinyaku
- Treatment: Exondys 51 · Sarepta
- Treatment: Vyondys 53 · Sarepta
- Treatment: Amondys 45 · Sarepta
- Treatment: Emflaza · Ptc Therap
- All diseases: Browse the disease index · Disease landscape hub
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing