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.
Raymond A. Huml, MS, DVM, RAC, is Executive Director, Strategic Drug Development, in the Biosimilars Center of Excellence at Quintiles. He was recently the author of the SpringerBrief, Pharmaceutical Competitive Intelligence for the Regulatory Affairs Professional. He has recruited several prominent MD's to contribute chapters to Muscular Dystrophy: A Concise Guide.
1. Introduction to Muscular Dystrophy.- 2.Muscular Dystrophy: Historical Background and Types.- 3.FSHD: The Most Common Type of MD?.- 4.Duchenne and Becker Muscular Dystrophies: Underlying Genetic and Molecular Mechanisms.- 5.An Overview of the Muscular Dystrophies: Underlying Genetic and Molecular Mechanisms.- 6.Transition from Childhood to Adult in Patients with Muscular Dystrophy.- 7.Overview of Current Treatments for Muscular Dystrophy.- 8.Physical Therapy and Orthotic Devices for Patients with Muscular Dystrophy.- 9.Orthopedic Care of Children with Muscular Dystrophy.- 10.Global Regulatory Landscape.- 11.Key Challenges to the Approval of Products to Treat Patients with Muscular Dystrophy.- 12.Pharmaceutical Products and Non-Pharmaceutical Interventions as Potential Treatments for Patients with Muscular Dystrophy.- 13.US Patient Advocacy Groups.- 14.Global and National Patient Registries.- 15.Summary.- Index.