Table 10 Current treatment guidelines for DMD

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