North East and North Cumbria
ICS Formulary
 
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10 Musculoskeletal and joint diseases
10-02-01 Drugs used in neuromuscular disorders

Ataluren Translarna®
Formulary
  • Approved as an option for treating Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene in people 2 years and over who can walk - in line with NICE HST22.
Link  NICE HST22: Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene

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Ravulizumab Ultomiris®
Non Formulary
  • 300mg/30ml and 110mg/11ml concentrate for solution for infusion
  • Not recommended for treating generalised myasthenia gravis in adults in line with NICE
  • Not recommended for treating AQP4 antibody-positive neuromyelitis optica spectrum disorder in adults in line with NICE
Link  NICE TA940: Ravulizumab for treating generalised myasthenia gravis (terminated appraisal)
Link  NICE TA941: Ravulizumab for treating AQP4 antibody-positive neuromyelitis optica spectrum disorder (terminated appraisal)

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