The field of muscular dystrophies has expanded significantly with the discovery of the genetic defects and protein products underlying these disorders. New treatments such as antisense oligonucleotides and siRNAs and gene therapies are now in clinical application and in trials. As clinical trials increase, outcome measurement becomes very important, and more validated tools are being developed.
This book provides a comprehensive review of these disorders. Clinical features, diagnostic testing including genetic testing of these disorders, the basis and utility of genetic testing, and the basis of genetic therapies are all covered in detail.
This book will provide neuromuscular neurologists, general neurologists, and neuromuscular fellows with a much needed update in the field.
Pushpa Narayanaswami, MD
Associate Professor of Neurology, Harvard Medical School
Vice- Chair, Clinical Operations
Department of Neurology
Beth Israel Deaconess Medical Center
Neuroloy TCC-8, BIDMC
330 Brookline Avenue
Boston MA 02215
Teerin Liewluck, MD, FAAN
Consultant, Division of Neuromuscular Medicine and Muscle Laboratory, Department of Neurology
Associate Professor of Neurology,
Mayo Clinic College of Medicine
200 First Street SW
Rochester, MN 55905