• 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.

8.86 mg/mL oral suspension

Approved in line with NICE guidance for treating Duchenne muscular dystrophy in people 6 years and over 

• Approved for the treatment of myotonia in patients with non-dystrophic myotonic disorders in line with NICE and NHSE commissioning policy (SSC2001).

• 12mg solution for injection

• Approved as an option for treating 5q spinal muscular atrophy (SMA) types 1, 2 or 3 in line with NICE.

• 0.75mg/1ml oral solution
• Approved for treating spinal muscular atrophy in line with NICE
• To be provided by Newcastle Hospitals homecare service only 

560 mg/4 ml solution for injection

Approved in line with NICE guidance for antibody-positive generalised myasthenia gravis

• 40 mg/ml oral suspension
• Approved for treating Duchenne muscular dystrophy in people 4 years and over in line with NICE

• Not recommended for treating antibody-positive generalised myasthenia gravis in line with NICE

• 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

• Not recommended for preventing relapses in neuromyelitis optica spectrum disorder in line with NICE