Therapeutic advances in muscular dystrophy.
The muscular dystrophies comprise a heterogeneous group of genetic disorders that produce progressive skeletal muscle weakness and wasting. There has been rapid growth and change in our understanding of these disorders in recent years, and advances in basic science are being translated into increasing numbers of clinical trials. This review will discuss therapeutic developments in 3 of the most common forms of muscular dystrophy: Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and myotonic dystrophy. Each of these disorders represents a different class of genetic disease (monogenic, epigenetic, and repeat expansion disorders), and the approach to therapy addresses the diverse and complex molecular mechanisms involved in these diseases. The large number of novel pharmacologic agents in development with good biologic rationale and strong proof of concept suggests there will be an improved quality of life for individuals with muscular dystrophy.
Center for Genetic Muscle Disorders, Kennedy Krieger Institute, Baltimore, MD; Departments of Neurology, Johns Hopkins School of Medicine, Baltimore, MD.
Doris G
DG
Leung
Kathryn R
KR
Wagner
D015316:Genetic Therapy; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000235:genetics; D020391:Muscular Dystrophy, Facioscapulohumeral / Q000188:drug therapy* / Q000235:genetics; D009223:Myotonic Dystrophy / Q000188:drug therapy* / Q000235:genetics