Non-Invasive Monitoring of Muscle Response to Disease Progression and Therapeutic Interventions in Duchenne Muscular Dys...

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Material Information

Title:
Non-Invasive Monitoring of Muscle Response to Disease Progression and Therapeutic Interventions in Duchenne Muscular Dystrophy Using MRI and MRS
Physical Description:
1 online resource (152 p.)
Language:
english
Creator:
Arpan, Ishu
Publisher:
University of Florida
Place of Publication:
Gainesville, Fla.
Publication Date:

Thesis/Dissertation Information

Degree:
Doctorate ( Ph.D.)
Degree Grantor:
University of Florida
Degree Disciplines:
Rehabilitation Science
Committee Chair:
VANDENBORNE,KRISTA H ELVIRE
Committee Co-Chair:
BYRNE,BARRY JOHN
Committee Members:
SHECHTMAN,ORIT
WALTER,GLENN A

Subjects

Subjects / Keywords:
biomarker -- dmd -- mri -- mrs
Rehabilitation Science -- Dissertations, Academic -- UF
Genre:
Rehabilitation Science thesis, Ph.D.
bibliography   ( marcgt )
theses   ( marcgt )
government publication (state, provincial, terriorial, dependent)   ( marcgt )
born-digital   ( sobekcm )
Electronic Thesis or Dissertation

Notes

Abstract:
Duchenne muscular dystrophy DMD is an inherited neuromuscular disorder characterized by progressive muscle degeneration and weakness. Boys with DMD experience gradual disability; with loss of ambulation occurring at approximately 8-12 years of age and death usually occurs early in the third decade of life. Despite the poor prognosis for patients with muscular dystrophy, therapeutic interventions have been lacking, and outcome measures for clinical trials have been limited to measures of muscle function and quality of life, serum biomarkers of muscle breakdown and muscle biopsies. Unfortunately, muscle biopsy is an invasive procedure, which limits its application for repeated measurements from the same area particularly in children. Strength and functional tests are important clinical tools for monitoring the disease progression in muscle groups. However, these tests are dependent on subject motivation and compliance, which can be particularly challenging in young children. Furthermore, six-minute walk test, a primary outcome measure in recent clinical trials, has been criticized for being not able to demonstrate functional decline in motor performance in 7-year-old boys with DMD of age. Since young boys with DMD are more likely to get maximum benefits from potential therapeutic interventions, there is a desperate need for sensitive and reliable biomarkers that can characterize skeletal muscle involvement in young boys with DMD and can facilitate the rapid translation of promising new therapies from preclinical studies to clinical trials. In the past few decades, magnetic resonance imaging MRI and spectroscopy MRS have become important non-invasive tools for studying skeletal muscle structure and composition enhancing our understanding of muscle physiology and pathophysiology. This doctoral dissertation presents results of three studies that demonstrate the ability of various MR techniques to monitor skeletal muscle response to disease progression as well as therapeutic interventions with DMD
General Note:
In the series University of Florida Digital Collections.
General Note:
Includes vita.
Bibliography:
Includes bibliographical references.
Source of Description:
Description based on online resource; title from PDF title page.
Source of Description:
This bibliographic record is available under the Creative Commons CC0 public domain dedication. The University of Florida Libraries, as creator of this bibliographic record, has waived all rights to it worldwide under copyright law, including all related and neighboring rights, to the extent allowed by law.
Statement of Responsibility:
by Ishu Arpan.
Thesis:
Thesis (Ph.D.)--University of Florida, 2014.
Local:
Adviser: VANDENBORNE,KRISTA H ELVIRE.
Local:
Co-adviser: BYRNE,BARRY JOHN.
Electronic Access:
RESTRICTED TO UF STUDENTS, STAFF, FACULTY, AND ON-CAMPUS USE UNTIL 2016-05-31

Record Information

Source Institution:
UFRGP
Rights Management:
Applicable rights reserved.
Classification:
lcc - LD1780 2014
System ID:
UFE0046559:00001