Skip to main content

ataluren (Translarna®)

 

Status: Recommended

The National Institute for Health and Care Excellence (NICE) published advice in July 2016 in relation to ataluren (Translarna®) for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older. At their meeting on 13 July 2016 AWMSG supported the adoption and implementation of this advice within NHS Wales. Welsh Government has subsequently agreed that the NICE HST advice for ataluren (Translarna®) should be implemented within NHS Wales. Refer to HST22 Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene for full guidance on NICE recommendations, including any specific restrictions on the use of the technology.

Medicine details

Medicine name ataluren (Translarna®)
Formulation 125 mg, 250 mg and 1000 mg granules for oral suspension
Reference number 940
Indication

Treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older

Company PTC Therapeutics Ltd
BNF chapter Musculoskeletal & joint diseases
Submission type N/A
Status Recommended
Date of issue 19/08/2016
NICE guidance

HST22 Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene

Commercial arrangement MAA
Further information

See: WHSSC Specialised Services Policy: CP118 Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene

 

Follow AWTTC: