Limb girdle muscular dystrophy: a prospective follow-up study of functional impairment

This 6-year prospective follow-up study evaluated the rate of clinical disease progression in 19 of 20 previously reported patients with strictly selected limb girdle muscular dystrophy. There was no significant deterioration in muscle strength (assessed by manual muscle testing). An activities of daily living (ADL) scale showed significant functional deterioration in 30% of patients with predominant involvement in a single functional domain variable between patients, and determined by the patient's previous level of function. A functional grading system showed that 70% of patients had deteriorated at least one grade in the arms and/or legs. Deterioration of the ADL score correlated with the loss of functional grades. We conclude that assessment of functional ability may be more sensitive to detect clinical disease progression compared to testing of muscle strength. The clinical parameter that correlates best with the activity of the underlying disease process is not determined. Tests for strength and functional impairment are complementary. Patients expressed appreciation that functional disability was addressed during follow-up visits.