Functional evaluation of Duchenne muscular dystrophy: proposal for a protocol

Abstract

A protocol for the evaluation of functional activities in subjects with Duchenne muscular dystrophy (DMD) was designed. The aim of our study was to define objective clinical criteria for the evaluation both of the clinical status of the patient and of the natural history of the illness itself. A protocol with such criteria is particularly necessary when testing the efficacy of treatment. 43 still-ambulant children with DMD between the ages of 3.10 yr and 10.4 yr were examined. Of this number 19 children were evaluated every 4 months over a period of 12 months; of these 14 formed part of a randomized double blind trial with L-carnitine (1.2-1.8 g/day) versus placebo.