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Showing 1 to 8 of 8 results for ataluren
Evidence-based recommendations on ataluren (Translarna) for Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in people 2 years and over who can walk.
Givinostat for treating Duchenne muscular dystrophy in people 6 years and over [ID6323]
In development [GID-TA11373] Expected publication date: TBC
Ataluren for treating cystic fibrosis in people with a CFTR gene nonsense mutation [ID955]
Discontinued [GID-TA11262]
Mexiletine for treating the symptoms of myotonia in non-dystrophic myotonic disorders (TA748)
Evidence-based recommendations on mexiletine (Namuscla) for treating the symptoms of myotonia in adults with non-dystrophic myotonic disorders.
Asfotase alfa for treating paediatric-onset hypophosphatasia (HST23)
Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in babies, children, young people and adults.
This guidance has been updated and replaced by NICE highly specialised technologies guidance 22.
NICE recommends routine funding for Duchenne muscular dystrophy gene therapy ataluren
NICE has today (22 February 2023) published final guidance which recommends ataluren (also called Translarna and made by PTC Therapeutics) for treating Duchenne muscular dystrophy (DMD) caused by a nonsense mutation.