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Full Description
Cardiorespiratory Aspects of Duchenne Muscular Dystrophy: A New Approach for Clinicians, Researchers and Regulators is designed to address key challenges in the field through a cardiorespiratory perspective on clinical care, drug development, and regulatory approval of new drugs. The aim is to review the current status, including achievements and limitations, and to make recommendations for advancing the field. This unique focus contrasts with existing literature, which often lists standards of care without addressing the practical challenges of clinical care, the need for better drugs through improved study methodologies, or the issues in current regulatory pathways. The book's cardiorespiratory focus on DMD, its approach to subtopics, critiques of methodology and regulatory pathways, and ideas for improvement are all novel. This perspective is designed to provide important and interesting information to a variety of stakeholders, including clinicians, researchers, and regulators. Clinicians will find not only standards of care but also strategies to overcome the challenges they face in enacting those standards. Researchers will gain insights into the crucial importance of cardiorespiratory outcome measures in studies of new DMD drugs, an area that is currently under-studied. Regulators will benefit from an examination of potential flaws in the current regulatory approval process, which can result in the approval of drugs that may be marginally effective or ineffective. The book will link critical reviews to proposals for better study methodologies and improvements in the regulatory process. This comprehensive approach aims to enhance patient survival by focusing on the key determinants of cardiorespiratory health in DMD.
Contents
Part 1. Clinical Topics
1. Duchenne muscular dystrophy. Genetics, Pathophysiology, Clinical Complication, and Overview of Multi-disciplinary Care
2. Respiratory Management and Standards of Care
3. Cardiac Management and Standards of Care
4. Practical Solutions to Common Clinical Problems
5. Anesthetic Strategies and Challenges
6. Cardiorespiratory Palliative Care
7. The Era of Emerging Genetic and Molecular Therapies
Part 2. Research Topics
8. Drug Study Methodologies. Potential Flaws in Outcome Measures
Part 1. Threshold Forced Vital Capacity Levels, Use of Historical Controls, and the Canadian Neuromuscular Data Registry
9. Drug Study Methodologies. Potential Flaws in Outcome Measures
Part 3. FVC % Predicted versus Rideau Plots
10. Methodologies. Cardiorespiratory Phenotypic Variability and the Need for Phenotypically Homogeneous Study Populations
11. Methodologies. New Outcome Proposals Based on Rideau Plots
12. Methodologies. Cardiorespiratory Phenotypic Discordance and the Importance of Cardiac Function in Determining Survival
13. The Need for Cardiac Outcome Measures
Part 4. Regulatory Topics
14. Problems With The Accelerated Approval Pathway of FDA and EMA
15. Case Studies of Current Regulatory Approvals. How the Accelerated Pathway and Suboptimal Study Methodologies Facilitate Potentially Ineffective Drugs
16. Conclusions and Future Directions
