03612nam 22006015 450 991030018110332120200701222803.03-319-17362-610.1007/978-3-319-17362-7(CKB)3710000000449534(EBL)3567780(SSID)ssj0001534784(PQKBManifestationID)11824413(PQKBTitleCode)TC0001534784(PQKBWorkID)11495834(PQKB)11408486(DE-He213)978-3-319-17362-7(MiAaPQ)EBC3567780(PPN)187684677(EXLCZ)99371000000044953420150716d2015 u| 0engur|n|---|||||txtccrMuscular Dystrophy A Concise Guide /edited by Raymond A. Huml1st ed. 2015.Cham :Springer International Publishing :Imprint: Springer,2015.1 online resource (206 p.)Description based upon print version of record.3-319-17361-8 Includes bibliographical references at the end of each chapters and index.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.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.Neurology PharmacotherapyMedicineNeurologyhttps://scigraph.springernature.com/ontologies/product-market-codes/H36001Pharmacotherapyhttps://scigraph.springernature.com/ontologies/product-market-codes/H69000Medicine/Public Health, generalhttps://scigraph.springernature.com/ontologies/product-market-codes/H00007Neurology .Pharmacotherapy.Medicine.Neurology.Pharmacotherapy.Medicine/Public Health, general.610Huml Raymond Aedthttp://id.loc.gov/vocabulary/relators/edtBOOK9910300181103321Muscular Dystrophy2245862UNINA