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ataluren (Translarna®)

Reference No. 3911

Publication date:

Appraisal information

ataluren (Translarna®) 125 mg granules for oral suspension
ataluren (Translarna®) 250 mg granules for oral suspension
ataluren (Translarna®) 1,000 mg granules for oral suspension

Company: PTC Therapeutics Ltd
BNF category: Musculoskeletal and joint diseases
NMG meeting date: 06/02/2019
AWMSG meeting date: 13/03/2019
Submission Type: Limited Submission
Status: Recommended
Advice No: 0419
Ratification by Welsh Government: 28/03/2019

Current Progress

Ratification by
Welsh Government

AWMSG advice

Ataluren (Translarna®) is recommended for use within NHS Wales for the 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. This recommendation applies only in circumstances where the approved Patient Access Scheme (PAS) is utilised or where the list/contract price is equivalent or lower than the PAS price.
Final Appraisal Recommendation (FAR)
AWMSG Secretariat Appraisal Report (ASAR)