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Table 10 Current treatment guidelines for DMD

From: The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review

Ref Guideline title Treatment type Further details of treatment type Population type to which recommendation applies Considerations Summary of recommendations Other Guideline comments
Bushby(d), 2010 [6] Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management Deflazacort Start on 0 · 9 mg/kg/day Age > =2, plateau or decline Consider as first line when pre-existing weight and/or behavioural issues Highly recommended for age > =6 and decline in age 2–6 Consider age, function (improving, plateau, declining), pre-existing risk factors, physician relationship with family. Ensure immunisation schedule is complete before initiating GCs. Alternative dosing strategies are also provided.
Prednisone Start on 0 · 75 mg/kg/day First line unless pre-existing weight and/or behavioural issues favour deflazacort Highly recommended for age > =6 and decline in age 2–5
Birnkrant, 2010 [59] The Respiratory Management of Patients With Duchenne Muscular Dystrophy Respiratory management Diagnostic testing, prevention, treatment, management under surgery All DMD All DMD Recommendations on necessary equipment, procedures, tests, and diagnostic evaluations. It also provides a structured approach to the assessment and management of the respiratory complications of DMD, emphasising the assessment of hypoventilation and the identification of specific thresholds of forced vital capacity (FVC), peak cough flow, and maximum expiratory pressure NR
Kinnett, 2015 [11] A Simplified Guide to Comprehensive Care for Muscular Dystrophy Multi-faceted Diagnosis, support networks, corticosteroids, cardiac, assessment, rehabilitation, respiratory management, mental health, self help All DMD All DMD Treatment recommendations include early start of corticosteroids, discussion of benefits and side effects by age of 3, evaluation of efficacy, discussion of longer term rationale for corticosteroid treatment. Other health maintenance strategies are promoted. NR