Lancashire and South Cumbria
Formulary
Formulary
10 Musculoskeletal and joint diseases
This chapter of the formulary is under continual development, please let the team know if you have any comments about the contents: mlcsu.lscformulary@nhs.net.
For more information about the formulary development process, please see: https://www.lancsmmg.nhs.uk/lancashire-and-south-cumbria-icb-formulary-development/
10-02 Drugs used in neuromuscular disorders
| Ataluren Translarna® |
|
Formulary
|
Granules for oral suspension 1000mg, 125mg, 250mg Ataluren for treating Duchenne muscular dystrphy with a nonsense mutation in the dystrophin gene (replaces HST3) - likely to be tertiary centre only |
 NICE:Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene |
|
| Nusinersen Spinraza® |
|
Formulary
|
Solution for injection vials 12mg/5ml |
|
NICE TA588: Nusinersen for treating spinal muscular atrophy |
|
|
| Onasemnogene abeparvovec Zolgensma® |
|
Formulary
|
2 × 1013 vector genomes/mL solution for infusion Tertiary centre |
|
NICE: Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy NICE: Onasemnogene abeparvovec for treating spinal muscular atrophy |
|
|
| Risdiplam Evrysdi® |
|
Formulary
|
Powder for oral solution 0.75mg/mL |
|
NICE TA755: Risdiplam for treating spinal muscular atrophy |
|
|
Links
LSCMMG: Gastroenterology Biologics Pathway- LSCMMG: Primary Care Constipation Guidelines
- MHRA: Antiepileptic drugs in pregnancy: updated advice following comprehensive safety review
- NICE CG147: Diverticular disease: diagnosis and management
- NICE CG184 Gastro-oesophageal reflux disease and dyspepsia in adults: investigation and management
- NICE CG61: Irritable bowel syndrome in adults: diagnosis and management
- NICE HST15: Onasemnogene abeparvovec for treating spinal muscular atrophy
- NICE HST24: Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy
- NICE NG1: Gastro-oesophageal reflux disease: recognition, diagnosis and management in children and young people
- NICE NST22 Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene
- NICE TA588: Nusinersen for treating spinal muscular atrophy
- NICE TA588: Nusinersen for treating spinal muscular atrophy
- NICE TA755: Risdiplam for treating spinal muscular atrophy
- NICE TA755:Risdiplam for treating spinal muscular atrophy
- NICE: Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy
- NICE: Onasemnogene abeparvovec for treating spinal muscular atrophy
- NICE:Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene