10.2. Drugs used in neuromuscular disorders
10.2.1. Drugs that enhance neuromuscular transmission
Restrictions:
Restricted to specialist initiation.
Prescribing Notes:
This medication is used for myasthenia gravis.
10.2.2. Skeletal muscle relaxants
Prescribing Notes:
Available as quinine sulphate and quinine bisulphate tablets, oral quinine is used primarily for nocturnal leg cramps. Patients should be reviewed regularly to establish benefit.
Restrictions: Baclofen injection is restricted to use in specialist units only. Slow withdrawal of baclofen over one to two weeks is recommended.
Restrictions:
Restricted to specialist initiation. Restricted to recommendation by designated specialists.
Restrictions:
Restricted to specialist use.
Prescribing Notes:
Patients commencing treatment should be reviewed via a formal assessment conducted by the specialist service at 4 weeks. Treatment should be discontinued at that point if a clinically significant improvement is not seen. Patients should also continue to be reviewed regularly thereafter to ensure continuing benefit.
10.2.3. Other neuromuscular drugs
Restrictions:
Restricted to specialist use only.
Prescribing Notes:
Ataluren may be prescribed within the ultra-orphan pathway while further evidence on its effectiveness is generated. In 2025, the SMC will evaluate the medicine for routine use in NHS Scotland. This medicine is part of the national Ultra-Orphan Risk Share Scheme.
Restrictions:
Restricted to specialist use for the symptomatic treatment of myotonia in adults with non-dystrophic myotonic disorders.
Prescribing Notes:
For other indications please see section 2.3.2
Restrictions:
Restricted to specialist use only for the treatment of type II and III 5q spinal muscular atrophy (SMA).
Prescribing Notes:
- This therapy is available via the Scottish Government Ultra-orphan pathway with reassessment by SMC following 3 years of data collection. Appropriate paperwork needs completing before prescribing to ensure registration on the national risk sharing scheme.
- SMA type I is already accepted for use by SMC and is available in the GGC Paediatric Formulary.
Restrictions:
Restricted to specialist use only in the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA type 1, type 2 or type 3 or with one to four SMN2 [survival of motor neuron 2] copies.
Prescribing Notes:
Available via the Ultra Orphan Pathway with reassessment by SMC following 3 years of data collection. Appropriate paperwork needs completing before prescribing to ensure registration on the national risk sharing scheme.
Restrictions:
Restricted to specialist use for treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older.