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ataluren (Translarna®)


Reference No. 3911


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: In progress
Advice No: Not available
Ratification by Welsh Government: Not available

Current Progress


Submission
received
NMG
meeting
AWMSG
meeting
Ratification by
Welsh Government

Indication

Treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years to less than 5 years
AWMSG Secretariat Appraisal Report (ASAR)
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Preliminary Appraisal Recommendation (PAR)
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Company Response to the Preliminary Appraisal Recommendation (CR PAR)
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