Muscular Dystrophy : A Concise Guide

This practical and concise guide offers an overview of muscular dystrophy's complicated features, treatment options and general resources. New treatments and a greater understanding of proteins and structures associated with MD are discussed along with long term patient care. Also included are clinical and developmental challenges within the current regulatory landscape and recent scientific and clinical advances. __Muscular Dystrophy__ offers clinicians, researchers, pharmaceutical executives and patient advocacy groups an easy-to-read reference that provides the necessary perspectives of the care giver and patient. Front Matter....Pages i-xxiv Introduction to Muscular Dystrophy....Pages 1-3 Muscular Dystrophy: Historical Background and Types....Pages 5-7 FSHD: The Most Common Type of Muscular Dystrophy?....Pages 9-19 Duchenne and Becker Muscular Dystrophies: Underlying Genetic and Molecular Mechanisms....Pages 21-35 An Overview of the Other Muscular Dystrophies: Underlying Genetic and Molecular Mechanisms....Pages 37-53 Transition from Childhood to Adult in Patients with Muscular Dystrophy....Pages 55-64 Overview of Current Treatments for Muscular Dystrophy....Pages 65-71 Physical Therapy and Orthotic Devices....Pages 73-104 Orthopaedic Management of the Child with Muscular Dystrophy....Pages 105-117 Global Regulatory Landscape....Pages 119-129 Key Challenges to the Approval of Products to Treat Patients with Muscular Dystrophy....Pages 131-144 Pharmaceutical Products and Non-pharmaceutical Interventions as Potential Treatments for Patients with Muscular Dystrophy....Pages 145-156 U.S. Patient Advocacy Groups....Pages 157-167 Global and National Patient Registries....Pages 169-174 Summary....Pages 175-179 Back Matter....Pages 181-191 This practical and concise guide offers an overview of muscular dystrophy's (MD) complicated features, treatment options and general resources. New treatments and a greater understanding of proteins and structures associated with MD are discussed along with long-term patient care. Also included are clinical and developmental challenges within the current regulatory landscape and recent scientific and clinical advances. Muscular Dystrophy: A Concise Guide offers clinicians, researchers, pharmaceutical executives and patient advocacy groups an easy-to-read reference that provides the necessary perspectives of the care giver and patient