Lancashire and South Cumbria
Formulary
Formulary
10 Musculoskeletal and joint diseases
10-02 Drugs used in neuromuscular disorders
| Ataluren Translarna® |
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Formulary
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Granules for oral suspension 125mg, 250mg, 1000mg Tertiary Centre Only. |
 NICE HST22:Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene |
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| Nusinersen Spinraza® |
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Formulary
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Solution for injection vials 12mg/5ml Tertiary Centre Only. |
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MHRA: Nusinersen (Spinraza▼): reports of communicating hydrocephalus; discuss symptoms with patients and carers and investigate urgently NICE TA588: Nusinersen for treating spinal muscular atrophy |
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| Onasemnogene abeparvovec Zolgensma® |
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Formulary
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2 × 1013 vector genomes/mL solution for infusion Tertiary Centre Only. |
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NICE HST15: Onasemnogene abeparvovec for treating spinal muscular atrophy NICE HST24: Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy |
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| Risdiplam Evrysdi® |
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Formulary
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Powder for oral solution 0.75mg/mL, Tablet 5mg Tertiary Centre Only. |
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NICE TA755: Risdiplam for treating spinal muscular atrophy |
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Links
LSCMMG: Inflammatory Bowel Disease: High Cost Drugs Commissioning Pathway- LSCMMG: Primary Care Constipation Guidelines
- MHRA: Antiepileptic drugs in pregnancy: updated advice following comprehensive safety review
- MHRA: Corticosteroids: rare risk of central serous chorioretinopathy with local as well as systemic administration
- MHRA: Nusinersen (Spinraza▼): reports of communicating hydrocephalus; discuss symptoms with patients and carers and investigate urgently
- NHSE: Steroid Emergency Card to support early recognition and treatment of adrenal crisis in adults
- 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 HST15: Onasemnogene abeparvovec for treating spinal muscular atrophy
- NICE HST22:Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene
- NICE HST24: Onasemnogene abeparvovec for treating presymptomatic 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 TA1031: Vamorolone for treating Duchenne muscular dystrophy in people 4 years and over
- 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