Cardiorespiratory Aspects of Duchenne Muscular Dystrophy

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. - Advances the delivery of cardiorespiratory-focused care for individuals with DMD - Identifies potential flaws in current DMD drug study methodologies and recommends key improvements - Examines problems with the current DMD drug approval process and suggests changes aimed at ensuring that approved drugs are truly effective