Posted by Michael Wonder on 22 Feb 2023
Ataluren for the treatment of patients with Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene
22 February 2023 - This guidance updates and replaces NICE highly specialised technologies guidance HST3 on the use of ataluren for the treatment of patients with Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene.
Ataluren is recommended as an option for the treatment of ambulatory patients 2 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene.
It is recommended only if PTC Therapeutics provides ataluren according to the commercial arrangement.
