The genetic understanding of the muscular dystrophies has advanced considerably in the
last two decades. Over 25 different individual genes are now known to produce muscular …

Mutations in the genes encoding dystrophin and its associated proteins, the sarcoglycans,
lead to muscular dystrophy in humans and in mouse models. In the presence of identical …

Abstract Characterization of the mechanisms underlying various types of muscular dystrophy
has been an outstanding triumph of molecular biology. Increasing clarification of the …

Neuromuscular disorders are often caused by heterogeneous mutations in large, structurally
complex genes. Targeting compensatory modifier genes could be beneficial to improve …

The muscular dystrophies are a group of neuromuscular disorders associated with muscle
weakness and wasting, which in many forms can lead to loss of ambulation and premature …

New Findings What is the topic of this review? Genetic modifiers act on many different
physiological aspects of muscle disease. Understanding and identifying such modifiers is …

CHAPTER 1: CLINICAL PERSPECTIVE: PHENOTYPIC EXPRESSION IN MUSCULAR
DYSTROPHY Lewis P. Rowland Department of Neurology Columbia U Page 1 CHAPTER 1 …

There is no doubt that the study of the muscular dystrophies in recent years has been
exciting and rewarding. It has attracted the attention of many investigators of international …

Neuromuscular diseases, which encompass disorders that affect muscle and its innervation,
are highly heritable. Genetic diagnosis now frequently pinpoints the primary mutation …

Introduction: The material included in this compilation of therapeutic trials represents the
efforts of many scientists who sought to experiment with new approaches in an effort to alter …