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.
Sommario
È stato elaborato un protocollo di valutazione delle attività funzionali in soggetti affetti da distrofia muscolare Duchenne (DMD).
Scopo del nostro studio è stato quello di definire dei criteri clinici oggettivi di valutazione sia dello stato di malattia del paziente, sia della evoluzione naturale della malattia stessa. Esigenza questa particolarmente sentita ogni qualvolta si debba verificare l'efficacia o meno di un qualsiasi intervento terapeutico.
Sono stati esaminati 43 bambini con DMD, ancora deambulanti, di età compresa tra i 3.10 ed i 10.4 anni. Di questi, 19 bambini, di cui 14 facevano parte di un trial randomizzato in doppio cieco con L-carnitina verso placebo (1.5–1.8 g/die), sono stati valutati ogni 4 mesi per un periodo di 12 mesi.
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This work has been in part supported by a grant from the “Dino and Enzo Ferrari Foundation for Muscular Dystrophy Research”.
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Cornelio, F., Dworzak, F., Morandi, L. et al. Functional evaluation of Duchenne muscular dystrophy: Proposal for a protocol. Ital J Neuro Sci 3, 323–330 (1982). https://doi.org/10.1007/BF02043581
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DOI: https://doi.org/10.1007/BF02043581