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Muscular Dystrophy

A Concise Guide

  • Book
  • © 2015

Overview

Editors:
  1. Raymond A. Huml

    1. Biosimilars Center of Excellence, Quintiles, Inc., Durham, USA

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  • Provides an overview of muscular dystrophy's features and treatment options

  • Clinical and scientific advances are presented along with the regulatory landscape for muscular dystrophy

  • Concise and practical guide for healthcare professionals, pharmaceutical executives and patient advocacy groups

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Table of contents (15 chapters)

  1. Front Matter

    Pages i-xxiv
    Download chapter PDF

  2. Introduction to Muscular Dystrophy

    • Raymond A. Huml
    Pages 1-3

  3. Muscular Dystrophy: Historical Background and Types

    • Raymond A. Huml
    Pages 5-7

  4. FSHD: The Most Common Type of Muscular Dystrophy?

    • Raymond A. Huml, Daniel P. Perez
    Pages 9-19

  5. Duchenne and Becker Muscular Dystrophies: Underlying Genetic and Molecular Mechanisms

    • Jean K. Mah
    Pages 21-35

  6. An Overview of the Other Muscular Dystrophies: Underlying Genetic and Molecular Mechanisms

    • Jean K. Mah
    Pages 37-53

  7. Transition from Childhood to Adult in Patients with Muscular Dystrophy

    • Elba Yesi Gerena Maldonado, Kathryn R. Wagner
    Pages 55-64

  8. Overview of Current Treatments for Muscular Dystrophy

    • Zheng (Jane) Fan
    Pages 65-71

  9. Physical Therapy and Orthotic Devices

    • Laura E. Case
    Pages 73-104

  10. Orthopaedic Management of the Child with Muscular Dystrophy

    • Robert K. Lark, Elizabeth W. Hubbard
    Pages 105-117

  11. Global Regulatory Landscape

    • Raymond A. Huml
    Pages 119-129

  12. Key Challenges to the Approval of Products to Treat Patients with Muscular Dystrophy

    • Raymond A. Huml
    Pages 131-144

  13. Pharmaceutical Products and Non-pharmaceutical Interventions as Potential Treatments for Patients with Muscular Dystrophy

    • Raymond A. Huml
    Pages 145-156

  14. U.S. Patient Advocacy Groups

    • Meredith L. Huml
    Pages 157-167

  15. Global and National Patient Registries

    • Raymond A. Huml
    Pages 169-174

  16. Summary

    • Raymond A. Huml
    Pages 175-179

  17. Back Matter

    Pages 181-191
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Keywords

About this book

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.

Editors and Affiliations

  • Biosimilars Center of Excellence, Quintiles, Inc., Durham, USA

    Raymond A. Huml

About the editor

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.

 

Bibliographic Information

  • Book Title: Muscular Dystrophy

  • Book Subtitle: A Concise Guide

  • Editors: Raymond A. Huml

  • DOI: https://doi.org/10.1007/978-3-319-17362-7

  • Publisher: Springer Cham

  • eBook Packages: Medicine, Medicine (R0)

  • Copyright Information: Springer International Publishing Switzerland 2015

  • Softcover ISBN: 978-3-319-17361-0Published: 30 July 2015

  • eBook ISBN: 978-3-319-17362-7Published: 16 July 2015

  • Edition Number: 1

  • Number of Pages: XXIV, 191

  • Number of Illustrations: 13 b/w illustrations, 30 illustrations in colour

  • Topics: Neurology, Pharmacotherapy, Medicine/Public Health, general

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