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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| Givinostat Duvyzat® |
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Formulary
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Oral Suspension
Givinostat can be used as an option to treat Duchenne muscular dystrophy in people 6 years and over who are ambulant (able to walk or stand, with or without support) at the start of treatment.
Givinostat will be available via the Innovative Medicines Fund (IMF) from 8th May 2026 in line with these recommendations. |
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NICE GID -TA11373: Givinostat for treating Duchenne muscular dystrophy in people 6 years and over |
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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
Biosimilars policy statement- Lancashire and South Cumbria Infant Feeding Network - Diagnosis and Management of Milk Allergy and Intolerance Guideline
- 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 GID -TA11373: Givinostat for treating Duchenne muscular dystrophy in people 6 years and over
- 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