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Functional evaluation of Duchenne muscular dystrophy: proposal for a protocol
Italian Journal of Neurological Sciences
|December 1, 1982
Summary
This study developed a functional evaluation protocol for Duchenne muscular dystrophy (DMD) to assess patient status and disease progression. The protocol aids in evaluating treatments for this neuromuscular disorder.
Area of Science:
- Neurology
- Pediatrics
- Clinical Trials
Background:
- Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder.
- Objective clinical criteria are needed to evaluate DMD patient status and natural history.
- Standardized protocols are essential for assessing treatment efficacy in DMD.
Purpose of the Study:
- To design a protocol for evaluating functional activities in children with Duchenne muscular dystrophy.
- To establish objective clinical criteria for assessing DMD patient status and disease progression.
- To provide a framework for testing the efficacy of therapeutic interventions for DMD.
Main Methods:
- Development of a functional evaluation protocol for DMD.
- Examination of 43 ambulant children with DMD (ages 3.10–10.4 years).
- Longitudinal evaluation of 19 children over 12 months, with 14 participating in a L-carnitine versus placebo trial.
Main Results:
- A protocol for functional assessment in DMD was established.
- Objective criteria for evaluating DMD clinical status and natural history were defined.
- Data collected will inform future treatment efficacy studies.
Conclusions:
- The developed protocol provides objective measures for DMD functional assessment.
- This standardized approach is crucial for tracking disease progression and treatment outcomes in DMD.
- The protocol serves as a foundation for future clinical trials in Duchenne muscular dystrophy.