Duchenne muscular dystrophy
Treatment of Duchenne muscular dystrophy
Prednisolone
Prednisolone 5mg soluble tablets sugar free
Dose as per specialist.
Deflazacort
Deflazacort 6mg tablets
Dose as per specialist.
Prescribing Notes:
- Treatment of Duchenne muscular dystrophy should be initiated only on specialist advice.
- Prior to starting steroid treatment baseline investigations including DEXA and varicella status should be obtained.
- Regular monitoring of bone health, blood pressure, growth and weight is required with steroid treatment.
History Notes
28/11/2024
Updating 'Prednisolone 5mg soluble tablets' to sugar free preparation - ERFC Dec 2024.
09/11/2023
East Region Formulary content agreed.
Additional treatment of Duchenne muscular dystrophy with nonsense mutation
Prescribing Notes:
- The initial assessment of evidence for Ataluren for treatment of Duchenne muscular dystrophy with nonsense mutation (SMC2327), has been completed following the SMC ultra-orphan framework. An updated submission to, and reassessment from, SMC for routine use in NHS Scotland would be expected in 2024.
- Treatment with ataluren is licensed in those patients aged 2 years of age and over who are ambulant.
History Notes
09/11/2023
East Region Formulary content agreed.