Cardiorespiratory Aspects of Duchenne Muscular Dystrophy: New Perspectives and Controversies is designed to address key challenges in the field by providing a cardiorespiratory perspective on clinical care, drug development, and the regulatory approval of new drugs. The book discusses current achievements and limitations of care and addresses clinical challenges. It offers a detailed, critical examination of the drug development process and identifies potential flaws in current regulatory processes. Novel recommendations for improving study methodologies and regulatory decisions are discussed, with the goal of enabling more effective drugs to reach individuals with Duchenne Muscular Dystrophy (DMD).
This book is designed to provide important and interesting information to a variety of stakeholders, including clinicians, researchers, and regulators, as well as patients and their caregivers. Clinicians will find a summary of standards of care and strategies to overcome the challenges they are likely to face in enacting those standards. Researchers will gain insights into the crucial importance of cardiorespiratory outcome measures and find recommendations designed to improve study methodologies. Regulators will benefit from an examination of potential flaws in the current drug approval process and from proposed recommendations aimed at ensuring that approved drugs are truly effective. The goal of this comprehensive approach is to enhance the outcomes and survival of individuals with DMD by focusing on key determinants of cardiorespiratory health.
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Table of Contents
Part 1: Clinical Management1. Respiratory Management and Standards of Care
2. Cardiac Management and Standards of Care
3. The Sleep Complications of Duchenne Muscular Dystrophy
4. Challenges in Managing the Patient with Advanced Duchenne Muscular Dystrophy and an Upper Respiratory Infection
5. Practical Solutions to Common Respiratory Challenges
6. Anesthetic Strategies and Challenges
7. Palliative Care for Individuals with Duchenne Muscular Dystrophy
Part 2: Drug Development for DMD: New Perspectives and Controversies
8. Potential Flaws in the Use of Historical Controls and Threshold Measures of Pulmonary Function
9. Additional Flaws in use of FVC % Predicted as an Outcome Measure and Proposed Improvements Based on Rideau Plots
10. Cardiorespiratory Phenotypic Variability
11. Cardiorespiratory Phenotypic Discordance and the Need for Cardiac Outcome Measures
Part 3: Regulatory Approvals for DMD: New Perspectives and Controversies
12. Problems With the Accelerated Approval Pathway of FDA
13. Case Study of Eteplirsen
14. Case Study of Elevidys
15. The Post-Approval Journey of Eteplirsen
16. The Post-Approval Journey of Elevidys
Part 4: Afterword
17. Afterword
Authors
David J. Birnkrant Professor Emeritus of Pediatrics, Case Western Reserve University School of Medicine, Cleveland, OH, United States. Dr. Birnkrant is Professor Emeritus of Pediatrics at Case Western Reserve University School of Medicine and a board-certified clinician in pediatric pulmonology and sleep medicine.With over 25 years in the pediatric neuromuscular clinic, he has dedicated his career to caring for individuals with neuromuscular diseases, particularly focusing on the cardiorespiratory management of Duchenne muscular dystrophy (DMD).
He has developed patents for respiratory devices to improve ventilatory assistance and has published extensively on DMD, emphasizing survival prolongation, anesthetic management, noninvasive ventilation, new drug therapies, and cardiorespiratory phenotypic variability.
His recent work critiques DMD drug study methodologies and regulatory pathways. Dr. Birnkrant was the lead author of the 2018 "Care Considerations for Duchenne Muscular Dystrophy� in The Lancet Neurology, now a standard of care.
He serves on the Editorial Board of Pediatric Pulmonology, has consulted for the FDA, and is the 2024 recipient of the Margaret Pfrommer Endowed Memorial Lecture award in home-based mechanical ventilation.
