Management and clinical trials in Duchenne Dystrophy

Corrado Angelini, Elisabetta Tasca

Research output: Chapter in Book/Report/Conference proceedingChapter

Abstract

Several studies have demonstrated the advantages and feasibility of conducting multicenter therapeutic trials in Duchenne muscular dystrophy (DMD). As a first step, the natural history of the disease and the influence of placebo must be identified. Since a number of clinical parameters are used in such assessment, it is important that evaluators follow a rigorous, standardized protocol. A clinical trial should also be randomized and double-blind in order to minimize the influence of placebo bias on scoring methods. Twelve clinical centers in Italy, with the coordinating center in Padova, have developed a computer assisted protocol to be used for multicenter, double-blind, placebo controlled therapeutic trial in DMD (Angelini et al. 1984). This multicenter therapeutic network was the first of its kind to study a muscular dystrophy in Europe. This protocol which includes manual muscle testing, myometric recording, and functional testing, is a model for therapeutic trials and should enular dystrophy (DMD) gene; FIGE able a large number of potentially effective medications to be tested in DMD using a standardized format. One year of natural history has been completed in 131 DMD patients, allowing us to test two separate drugs versus placebo and dietary advice.

Original languageEnglish
Title of host publicationDuchenne Muscular Dystrophy: Symptoms, Management and Prognosis
PublisherNova Science Publishers, Inc.
Pages133-141
Number of pages9
ISBN (Print)9781634821971, 9781634821537
Publication statusPublished - Apr 1 2015

Keywords

  • Branched chain aminoacids
  • Deflazacort
  • DMD natural history
  • Fructose diet
  • Management

ASJC Scopus subject areas

  • Medicine(all)

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  • Cite this

    Angelini, C., & Tasca, E. (2015). Management and clinical trials in Duchenne Dystrophy. In Duchenne Muscular Dystrophy: Symptoms, Management and Prognosis (pp. 133-141). Nova Science Publishers, Inc..