Rehabilitation Medicine Research
Rehabilitation Medicine Research

Research initiatives of the Division of Rehabilitation Medicine take a variety of forms, including clinical, translational and basic science. An important aspect of our research has been a number of collaborative projects in gene therapy for Duchenne Muscular Dystrophy, working closely with Dr. Robert White, Ph.D., Professor of Biochemistry, Kansas City University of Medicine and Biosciences.

Our current areas of clinical, translational and basic science research include

  • Gene therapy in Duchenne muscular dystrophy
    • The identification of human retinal dystrophin as an isoform of skeletal muscle dystrophin has led to a multi-institutional effort to perform gene therapy experiments. Scientists are using retinal dystrophin in DMD skeletal muscle as a replacement therapy for this lethal muscle disease. Introducing retinal dystrophin by genetic manipulation in severe DMD mice models prevents severe muscle degeneration, dilated cardiomyopathy and kyphosis. Also, these mice models have demonstrated a normal lifespan. We accomplished this study with grant support from the March of Dimes and the Patton Memorial Trust.
  • Outcome measures and management strategies in obstetric brachial plexus injuries
    • Social Functioning in Youths with Obstetric Brachial Plexus Injury.
    • Efficacy of Botulinum Toxin in the Treatment of Obstetric Brachial Plexus Injuries, Katherine B. Richardson grant funded study.
    • A Survey Assessing the Management of Neonatal Brachial Plexus Palsy in 23 institutions.
  • Oral pharmacology spasticity management
    • Baclofen Efficacy and Safety Trials - Pediatric Pharmacokinetic and Pharmacodynamic Study of Oral Baclofen for the Treatment of Spasticity Associated with Cerebral Palsy, NICHD funded multi-center study.
  • Community participation and quality of life
    • Assessment of Growth in Independent Living Skills in the Adolescent/Young Adult with Spina Bifida Following a Transition Camp Experience (data analysis phase).
    • Using Motivational Interviewing to Encourage Urinary Catheterization in Children and Adolescents with Spina Bifida (data collection phase).
  • Botox Treatment Drug Trials
    • Botox Treatment in Pediatric Upper and Lower Limb Spasticity in collaboration with Allergan
    • Botox and Limb Flexor Synergy
  • Data Repositories
    • Natural History of Dystrophinopathy Patients: Correlate the severity of the dystrophin-deficient cardiomyopathy with dystrophin gene mutations and skeletal muscle function in collaboration with Nationwide Children's Hospital and MDA
    • Development of an Intraoperative Neurophysiologic Monitoring data repository

Research Highlights

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