Following a limited submission
AWMSG advice |
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| Status: Superseded | |
AWMSG ADVICE SUPERSEDED BY NICE GUIDANCE ISSUED JANUARY 2023. Refer to HST22 Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene for full guidance on NICE recommendations. |
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Medicine details |
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| Medicine name | ataluren (Translarna®) |
| Formulation | 125 mg granules for oral suspension, 250 mg granules for oral suspension and 1,000 mg granules for oral suspension |
| Reference number | 3911 |
| Indication | Treatment of Duchenne muscular dystrophy (DMD) resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years to less than 5 years |
| Company | PTC Therapeutics Ltd |
| BNF chapter | Musculoskeletal & joint diseases |
| Submission type | Limited |
| Status | Superseded |
| Advice number | 0419 |
| NMG meeting date | 06/02/2019 |
| AWMSG meeting date | 13/03/2019 |
| Ratification by Welsh Government | 28/03/2019 |
| Date of issue | 28/03/2019 |
| NICE guidance | |
| Commercial arrangement | CAA |
| Further information |
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