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.
1. Title of Book - p. i 2. Cover image - p. i 3. Title page - p. ii 4. Table of Contents - p. iii 5. Copyright - p. iv 6. Dedication - p. v 7. Contributors - p. xi 8. About the editor - p. xiii 9. Preface - p. xv 10. Part 1. Clinical management - p. P1 11. Chapter 1. Respiratory management and standards of care - p. 3 12. Chapter 2. Cardiac management and standards of care - p. 17 13. Chapter 3. Sleep complications of Duchenne muscular dystrophy - p. 29 14. Chapter 4. Challenges in managing the patient with advanced Duchenne muscular dystrophy and an upper respiratory infection - p. 45 15. Chapter 5. Practical solutions to common respiratory challenges - p. 55 16. Chapter 6. Anesthetic strategies and challenges - p. 63 17. Chapter 7. Palliative care for individuals with Duchenne muscular dystrophy - p. 77 18. Part 2. Drug development for DMD: New perspectives and controversies - p. P2 19. Chapter 8. Potential flaws in the use of historical controls and threshold measures of pulmonary function - p. 91 20. Chapter 9. Additional flaws in the use of FVC % predicted as an outcome measure and proposed improvements based on Rideau plots - p. 99 21. Chapter 10. Cardiorespiratory phenotypic variability - p. 105 22. Chapter 11. Cardiorespiratory phenotypic discordance and the need for cardiac outcome measures - p. 117 23. Part 3. Regulatory approvals for DMD: New perspectives and controversies - p. P3 24. Chapter 12. Problems with the accelerated approval pathway of the FDA - p. 123 25. Chapter 13. Case study of eteplirsen - p. 131 26. Chapter 14. Case study of Elevidys - p. 143 27. Chapter 15. The post-approval journey of eteplirsen - p. 153 28. Chapter 16. The post-approval journey of Elevidys - p. 161 29. Part 4. Afterword - p. P4 30. Chapter 17. Afterword - p. 175 31. Index - p. 179
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.
1. Title of Book - p. i 2. Cover image - p. i 3. Title page - p. ii 4. Table of Contents - p. iii 5. Copyright - p. iv 6. Dedication - p. v 7. Contributors - p. xi 8. About the editor - p. xiii 9. Preface - p. xv 10. Part 1. Clinical management - p. P1 11. Chapter 1. Respiratory management and standards of care - p. 3 12. Chapter 2. Cardiac management and standards of care - p. 17 13. Chapter 3. Sleep complications of Duchenne muscular dystrophy - p. 29 14. Chapter 4. Challenges in managing the patient with advanced Duchenne muscular dystrophy and an upper respiratory infection - p. 45 15. Chapter 5. Practical solutions to common respiratory challenges - p. 55 16. Chapter 6. Anesthetic strategies and challenges - p. 63 17. Chapter 7. Palliative care for individuals with Duchenne muscular dystrophy - p. 77 18. Part 2. Drug development for DMD: New perspectives and controversies - p. P2 19. Chapter 8. Potential flaws in the use of historical controls and threshold measures of pulmonary function - p. 91 20. Chapter 9. Additional flaws in the use of FVC % predicted as an outcome measure and proposed improvements based on Rideau plots - p. 99 21. Chapter 10. Cardiorespiratory phenotypic variability - p. 105 22. Chapter 11. Cardiorespiratory phenotypic discordance and the need for cardiac outcome measures - p. 117 23. Part 3. Regulatory approvals for DMD: New perspectives and controversies - p. P3 24. Chapter 12. Problems with the accelerated approval pathway of the FDA - p. 123 25. Chapter 13. Case study of eteplirsen - p. 131 26. Chapter 14. Case study of Elevidys - p. 143 27. Chapter 15. The post-approval journey of eteplirsen - p. 153 28. Chapter 16. The post-approval journey of Elevidys - p. 161 29. Part 4. Afterword - p. P4 30. Chapter 17. Afterword - p. 175 31. Index - p. 179
