The quest for a therapy for muscular dystrophy has been the driving force behind the past 40
years of advances in this field. Numerous results, such as the identification of satellite cells …

In the fast moving field of muscular dystrophy, therapeutic matters are now high on the
agenda. Despite little progress made in the understanding of the exact pathogenesis, hopes …

In the last few years, significant advances have occurred in the preclinical and clinical work
toward gene and cell therapy for muscular dystrophy. At the time of this writing, several trials …

Introduction: Muscular dystrophies are a heterogeneous group of genetic diseases
characterized by muscle weakness, wasting and degeneration. Cell therapy consists of …

Cell-based therapy for muscular dystrophies was initiated in humans after promising results
obtained in murine models. Early trials failed to show substantial clinical benefit, sending …

Experimental therapeutics of the muscular dystrophies has made impressive advances on
several fronts. Adeno-associated virus has emerged as the clear 'vector of choice'for muscle …

The muscular dystrophies are a heterogeneous group of over 40 disorders that are
characterised by muscle weakness and wasting. The most common are Duchenne muscular …

Muscular dystrophies are individually rare genetic disorders that cause much chronic
disability, affecting young children and adults. In the past 20 years, more than 30 genetic …

For the past decade the molecular geneticists have been telling us that gene therapy is just
round the corner. Each year the corner seems further away. Each new technical advance, for …

Research into therapeutic approaches for both recessive and dominant neuromuscular
disorders has made great progress over the past few years. In the field of gene therapy …