Ataluren
Orphan DrugEMA Approved
Contents
Description
Ataluren is a drug designed to treat genetic disorders caused by nonsense mutations. It promotes ribosomal read-through of premature stop codons, allowing for the production of a full-length, functional protein.
Indications & Therapeutic Use
Duchenne muscular dystrophy with nonsense mutation
Linked Diseases:
Global Availability (5 countries)
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Buy / Source Ataluren — GNH India
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Ataluren
| Generic Name | Ataluren |
| Brands | 1 brand available |
| Active Ingredient | Ataluren |
| Drug Class | Duchenne muscular dystrophy with nonsense mutation |
| Manufacturer | PTC Therapeutics |
| Dosage Forms | Powder for oral suspension |
| Medical Code | N07XX09 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Lead Time | 21 days |
| Reg. Status | EMA Approved |
| Clinical Trial | NCT03123962 |
| Countries | 5 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations6 Validated Nodes