10.2.3. Other neuromuscular drugs
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 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.


