Pharmacologic Intervention

The rehabilitation specialist may become involved in the prescription of pharmacologic agents, which affect the pathophysiology of various neuromuscular diseases. Evaluation of therapeutic efficacy for phar­macologic agents requires careful objective measure­ment of strength, with quantitative measurements, functional status using timed motor testing and the six-minute walk test, pulmonary function parameters, cardiac parameters, and patient-reported quality of life measures.

Corticosteroids such as prednisone and deflazacort may have an effect on the inflammatory component of the dystrophic myopathy and other disease pathways in DMD, slow the progression of the strength loss, pro­long ambulation by two years, reduce the occurrence of scoliosis, and slow the loss of pulmonary function (11-13,22-24,156). Alternative pulsed-dosing regimens, such as high-dose weekend administration (5 mg/kg/ day on both Saturday and Sunday) may decrease the side effects of weight gain and growth retardation with similar clinical efficacy. Deflazacort is an alter­nate corticosteroid with a potentially better side effect profile and equal efficacy to prednisone in DMD.

The identification of specific genes and the pro­tein products implicated in the pathogenesis of var­ious neuromuscular diseases provides hope that meaningful therapeutic interventions that target the defective genes will alter the natural history of many of these neuromuscular diseases. It is likely that tra­ditional rehabilitation approaches will need to be used adjunctively with newer pharmacologic interventions, molecular genetic-based therapies, and possibly gene therapy in the management of these conditions.

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