Therapeutic trials on progressive muscular dystrophy

Intern Med. 1992 Jul;31(7):841-6. doi: 10.2169/internalmedicine.31.841.


The special medical care in the National Sanatorium prolonged the life span of the patients with progressive muscular dystrophy from 15.8 years to 20.4 years over the last 20 years. Various new drug trials for muscular dystrophy have been implemented in the last 12 years in Japan. Bestatin and Loxistatin, protease inhibitors, showed definite improvement on dystrophic mice or hamsters, animal models of muscular dystrophy. However clinical application of these drugs failed to prove the effects on patients with Duchenne muscular dystrophy. The difficulty of clinical evaluation and judgement of effects in progressive neurological diseases is discussed.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Review

MeSH terms

  • Animals
  • Child
  • Child, Preschool
  • Cricetinae
  • Double-Blind Method
  • Drug Evaluation, Preclinical
  • Humans
  • Incidence
  • Japan / epidemiology
  • Leucine / analogs & derivatives*
  • Leucine / therapeutic use
  • Life Expectancy
  • Male
  • Mice
  • Muscular Dystrophies / drug therapy*
  • Muscular Dystrophies / epidemiology
  • Muscular Dystrophy, Animal / drug therapy
  • Prevalence
  • Protease Inhibitors / therapeutic use*
  • Single-Blind Method


  • Protease Inhibitors
  • Leucine
  • ubenimex
  • aloxistatin