Muscular Dystrophy in Children

It is not a treatment for the dystrophy. Disease-modifying care is genetic and pharmacological, and it belongs with a neuromuscular specialist. We state this because families travelling internationally are sometimes offered intensive therapy on the implication that it will change the disease course.

What therapy aims for is slower contracture development, better positioning, longer independent function, and equipment that fits the child rather than the other way round. These aims rest on expert consensus rather than large trials, which are rare in this condition 1.

Eccentric and high-resistance exercise can damage dystrophic muscle. Submaximal aerobic activity, swimming and cycling are generally well tolerated and are the mainstay. Exercise to exhaustion is avoided, and increasing weakness or dark urine after a session is a reason to stop and reassess.

Stretching, night splints and standing programmes address the ankle, knee and hip contractures that otherwise end walking earlier than the muscle weakness alone would.

Seating and posture become the priority, because scoliosis progresses quickly once a child stops walking and it compromises breathing. Powered mobility maintains independence and is introduced as an expansion of what the child can do, not as a defeat.

Upper limb function, communication and access to schooling are the goals that matter to the family at this stage, and occupational therapy leads that work.

In dystrophic muscle, eccentric and high-resistance exercise can accelerate damage. Submaximal aerobic activity and range maintenance are what is prescribed instead.

This runs against the usual instinct to strengthen, and getting it wrong causes harm — which is why an unsupervised gym programme is inappropriate here.

In Duchenne muscular dystrophy, the most common form in childhood, steroid medicines are the treatment with the strongest evidence 1 3. A Cochrane review of randomised trials found moderate-quality evidence that they improve muscle strength and function over the first year and strength for up to two years, while evidence that they prolong walking comes only from non-randomised studies 3. Weight gain and changes in behaviour are common side effects 3.

International care guidance links long-term steroid treatment with walking being lost later, better-preserved arm and breathing function and less need for spinal surgery 1. Newer genetic medicines each target particular mutations, so eligibility depends on the child’s genetic result; these decisions sit with a neuromuscular specialist, and physiotherapy continues alongside them 1.

Duchenne affects the heart muscle and the breathing muscles as well as the limbs, and heart and breathing complications are among the main causes of serious illness and death 2. Both can decline without obvious symptoms: breathing capacity can fall without breathlessness, and signs of heart failure are easily missed once a child uses a wheelchair 2.

International care guidance therefore advises an ECG and heart imaging at diagnosis and then at least once a year, and breathing tests from about age 5–6 2. After walking stops, breathing measurements are repeated at least every six months 2.