Anticholinesterases are used for myasthenia gravis Their muscarinic side effects, including bradycardia and an increase in sweating, salivary and gastric secretion and gastrointestinal and uterine motility, are all antagonised by atropine.

Pyridostigmine bromide

• Tablets 60mg (£17.37 = 200 tablets)

Indications

• Myasthenia gravis

Dose

• 30–120mg at suitable intervals throughout day, total daily dose 0.3–1.2g

Ataluren

• Granules for oral suspension 125mg, 250mg, 1000mg

Notes

1. NICE HST22: Ataluren (​Translarna​) ​is recommended​, within its marketing authorisation, 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, only if the company provides it according to the commercial arrangement (February 2023).

Nusinersen

• Solution for injection vials 12mg in 5ml

Notes

1. NICE TA588: Nusinersen (Spinraza) is recommended as an option for treating 5q spinal muscular atrophy (SMA), only when the criteria of the NICE TA are met (July 2019)
2. MHRA Drug Safety Update (September 2018): Nusinersen (Spinraza): reports of communicating hydrocephalus; discuss symptoms with patients and carers and investigate urgently

Onasemnogene abeparvovec

• 2 × 10¹³ vector genomes/ml solution for infusion

Notes

1. HST15: Onasemnogene abeparvovec for treating spinal muscular atrophy (SMA) (July 2021)
   1. Onasemnogene abeparvovec (Zolgensma) is recommended as an option for treating 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of type 1 SMA in babies, only if:
      • they are 6 months or younger, or
      • they are aged 7 to 12 months, and their treatment is agreed by the national multidisciplinary team
   2. It is only recommended for these groups if:
      • permanent ventilation for more than 16 hours per day or a tracheostomy is not needed
      • the company provides it according to the commercial arrangement
2. NICE HST24: Onasemnogene abeparvovec (Zolgensma) is recommended as an option for treating presymptomatic 5q spinal muscular atrophy (SMA) with a biallelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene in babies aged 12 months and under, only if the company provides it according to the commercial arrangement (April 2023).
   

Risdiplam

• Oral solution sugar free 750micrograms/ml

Notes

1. NICE TA755: Risdiplam (Evrysdi) is recommended as an option for treating 5q spinal muscular atrophy (SMA) in people of all ages with a clinical diagnosis of SMA types 1, 2 or 3 or with pre-symptomatic SMA and 1 to 4 SMN2 copies, only if the conditions of the managed access agreement are followed (December 2021, updated December 2023). 

Fampridine

Not routinely funded for multiple sclerosis. See NHS England Clinical Commissioning Policy Statement: Fampridine for Multiple Sclerosis