Development of a Lifespan-Based Novel Composite Person Reported Outcome Measure Using Data From the CINRG Duchenne Natural History Study

Abstract

Development of novel technologies and therapeutic agents to treat Duchenne muscular dystrophy (DMD) have increased interest by regulatory bodies such as the Food and Drug Administration in the development of clinically-meaningful study endpoints for clinical trials. There is a need for the development of person-reported outcome (PRO) instruments that target a broad range of developmental and functional ability while effectively evaluating treatment effects in clinical trials.

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Document Details

Document Type
Technical Report
Publication Date
Oct 01, 2018
Accession Number
AD1070351

Entities

People

  • Craig M. McDonald
  • Erik Henricson

Organizations

  • University of California

Tags

DTIC Thesaurus Topics

  • Age Groups
  • Clinical Trials
  • Composite Materials
  • Data Analysis
  • Data Sets
  • Department Of Defense
  • Electronic Mail
  • Health
  • Health Care
  • Health Services
  • History
  • Materials
  • Natural History
  • Personnel Management
  • Quality Of Life
  • Questionnaires
  • Simulations

Fields of Study

  • Medicine

Readers

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