LEADER 03612nam 22006015 450 001 9910300181103321 005 20200701222803.0 010 $a3-319-17362-6 024 7 $a10.1007/978-3-319-17362-7 035 $a(CKB)3710000000449534 035 $a(EBL)3567780 035 $a(SSID)ssj0001534784 035 $a(PQKBManifestationID)11824413 035 $a(PQKBTitleCode)TC0001534784 035 $a(PQKBWorkID)11495834 035 $a(PQKB)11408486 035 $a(DE-He213)978-3-319-17362-7 035 $a(MiAaPQ)EBC3567780 035 $a(PPN)187684677 035 $a(EXLCZ)993710000000449534 100 $a20150716d2015 u| 0 101 0 $aeng 135 $aur|n|---||||| 181 $ctxt 182 $cc 183 $acr 200 10$aMuscular Dystrophy $eA Concise Guide /$fedited by Raymond A. Huml 205 $a1st ed. 2015. 210 1$aCham :$cSpringer International Publishing :$cImprint: Springer,$d2015. 215 $a1 online resource (206 p.) 300 $aDescription based upon print version of record. 311 $a3-319-17361-8 320 $aIncludes bibliographical references at the end of each chapters and index. 327 $a1. 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. 330 $aThis 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. 606 $aNeurology  606 $aPharmacotherapy 606 $aMedicine 606 $aNeurology$3https://scigraph.springernature.com/ontologies/product-market-codes/H36001 606 $aPharmacotherapy$3https://scigraph.springernature.com/ontologies/product-market-codes/H69000 606 $aMedicine/Public Health, general$3https://scigraph.springernature.com/ontologies/product-market-codes/H00007 615 0$aNeurology . 615 0$aPharmacotherapy. 615 0$aMedicine. 615 14$aNeurology. 615 24$aPharmacotherapy. 615 24$aMedicine/Public Health, general. 676 $a610 702 $aHuml$b Raymond A$4edt$4http://id.loc.gov/vocabulary/relators/edt 906 $aBOOK 912 $a9910300181103321 996 $aMuscular Dystrophy$92245862 997 $aUNINA