
Cardiorespiratory Aspects of Duchenne Muscular Dystrophy
New Perspectives and Controversies
by David J. Birnkrant
1st Edition
Publisher: Academic Press
Book Details
| Print ISBN | 9780443415982 |
| eText ISBN | 9780443415999 |
| Publisher | Academic Press |
| Publishing Year | 2026 |
| Edition | 1st Edition |
| Language | English |
| Pages | 168 |
Cardiorespiratory Aspects of Duchenne Muscular Dystrophy, 1st Edition, edited by David J. Birnkrant, addresses clinical care, drug development, and regulatory approval pathways for Duchenne muscular dystrophy from a cardiorespiratory perspective. Published by Academic Press, this publication provides structured material intended for clinicians, researchers, regulators, patients, and caregivers.
The volume synthesizes clinical management themes to evaluate both achievements and limitations in care for individuals with Duchenne muscular dystrophy. It covers respiratory management, cardiac care standards, and sleep complications, while presenting practical solutions for common respiratory challenges. Additional clinical sections discuss anesthetic strategies and palliative care approaches designed to support patient management.
Beyond routine intervention, the text examines therapeutic assessment methodologies through case studies of Eteplirsen and Elevidys. It offers clinicians clear summaries of care standards and clinical management strategies, giving healthcare teams and regulatory specialists grounded reference material to evaluate cardiorespiratory outcomes.
Table of Contents
Chapter 1: Respiratory Management and Standards of Care
Chapter 2: Cardiac Management and Standards of Care
Chapter 3: The Sleep Complications of Duchenne Muscular Dystrophy
Chapter 4: Challenges in Managing the Patient with Advanced Duchenne Muscular Dystrophy and an Upper Respiratory Infection
Chapter 5: Practical Solutions to Common Respiratory Challenges
Chapter 6: Anesthetic Strategies and Challenges
Chapter 7: Palliative Care for Individuals with Duchenne Muscular Dystrophy
Chapter 8: Potential Flaws in the Use of Historical Controls and Threshold Measures of Pulmonary Function
Chapter 9: Additional Flaws in use of FVC % Predicted as an Outcome Measure and Proposed Improvements Based on Rideau Plots
Chapter 10: Cardiorespiratory Phenotypic Variability
Chapter 11: Cardiorespiratory Phenotypic Discordance and the Need for Cardiac Outcome Measures
Chapter 12: Problems With the Accelerated Approval Pathway of FDA
Chapter 13: Case Study of Eteplirsen
Chapter 14: Case Study of Elevidys
Chapter 15: The Post-Approval Journey of Eteplirsen
Chapter 16: The Post-Approval Journey of Elevidys
Chapter 17: Afterword
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