Meet the editor

Florina Gisela Gaina, PhD, currently works at "Victor Babes" National Institute of Pathology, Bucharest, Romania. She received her PhD in Biology from the University of Bucharest, Romania, in 2009 with a thesis project based on the study of the proteins involved in muscular dystrophies. She is a research scientist working in the field of skeletal muscle. The primary focus of her research activities is on skeletal muscle regeneration. She

has been involved in a number of research projects funded by regional, national, and international public agencies. She is an author and/or co-author of more than twenty scientific papers and conference abstracts and three book chapters.

Contents

and Present Perspectives

*by Robin Warner*

*and Amelia Aranega*

Dystrophy

Dystrophies

*and Jonathan Dando*

**Preface XI**

**Chapter 1 1**

**Chapter 2 17**

**Chapter 3 27**

**Chapter 4 51**

**Chapter 5 63**

**Chapter 6 81**

Duchenne Muscular Dystrophy (DMD) Treatment: Past

Facioscapulohumeral Muscular Dystrophy: Genetics and Trials

*by Francisco Hernandez-Torres, Lara Rodriguez-Outeiriño, Lidia Matias-Valiente, Estefania Lozano-Velasco, Diego Franco* 

Role of Growth Factors and Apoptosis Proteins in Cognitive Disorder Development in Patients with Duchenne Muscular

Mesenchymal Stem Cells for Regenerative Medicine for

*by Ahmed Elhussieny, Ken'ichiro Nogami, Fusako Sakai-Takemura, Yusuke Maruyama, AbdElraouf Omar Abdelbakey, Wael Abou El-kheir,* 

The Impact of Payer and Reimbursement Authorities Evidence Requirements on Healthcare Solution Design for Muscular

*by Maximilian Lebmeier, Fleur Chandler, Josie Godfrey* 

*by Nahla O. Mousa, Ahmed Osman, Nagia Fahmy,* 

*Ahmed Abdellatif and Waheed K. Zahra*

miRNAs and Muscle Stem Cells

*by Mariia Georgievna Sokolova and Ekaterina Valentinovna Lopatina*

Duchenne Muscular Dystrophy

*Shin'ichi Takeda and Yuko Miyagoe-Suzuki*

## Contents


Preface

Muscular dystrophies are a group of genetic disorders characterized by progressive weakness and loss of muscle mass. Although more than 30 years have passed since the discovery of the first protein involved in a type of muscular dystrophy, there is still no cure for these conditions. In the last decades, with the improvement of existing molecular biology techniques and the development of new approaches, many efforts have been made to accelerate the disease diagnostic process, to better understand the molecular defects and mechanisms underlying the molecular

Consequently, the development of different effective therapeutic strategies that slow down the course of the disease and improve patient quality of life and mortal-

This book provides a comprehensive overview of the recent advances in the area of muscle diseases covering clinical manifestations, current diagnostic and therapeutic strategies, clinical trials, and their specific issues. In addition, this book updates the knowledge on mesenchymal stem/progenitor cells and miRNA, and discusses their therapeutic potential in regenerative medicine in a clear and concise manner.

We are very pleased to have had the opportunity to write this book on muscular dystrophy for IntechOpen, and we hope that this book will offer inspiration for young and experienced researchers to answer the many questions muscle pathology

I would like to thank Author Service Manager Romina Rovan for her patience and

**Florina Gisela Gaina**

Bucharest, Romania

Victor Babes National Institute of Pathology,

valuable advice throughout the preparation of this book.

pathogenesis involved in dystrophy conditions.

ity continues to be a priority for researchers.

raises.
