Therapeutics in duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a fatal disorder affecting approximately 1 in 3,500 live born males, characterized by progressive muscle weakness. Several different strategies are being investigated in developing a cure for this disorder. Until a cure is found, therapeutic and supportive care is essential in preventing complications and improving the afflicted child's quality of life. Currently, corticosteroids are the only class of drug that has been extensively studied in this condition, with controversy existing over the use of these drugs, especially in light of the multiple side effects that may occur. The use of nutritional supplements has expanded in recent years as researchers improve our abilities to use gene and stem cell therapies, which will hopefully lead to a cure soon. This article discusses the importance of therapeutic interventions in children with DMD, the current debate over the use of corticosteroids to treat this disease, the growing use of natural supplements as a new means of treating these boys and provides an update on the current state of gene and stem cell therapies.
Pediatric Muscular Dystrophy Association Clinic, University of California, San Francisco, USA. stroberj@neuropeds.ucsf.edu
Jonathan B
JB
Strober
D000305:Adrenal Cortex Hormones / Q000627:therapeutic use*; D000617:Aminoglycosides / Q000627:therapeutic use*; D000818:Animals; D000906:Antibodies / Q000627:therapeutic use; D015316:Genetic Therapy; D006801:Humans; D051379:Mice; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy / Q000601:surgery / Q000628:therapy*; D044623:Nutrition Therapy; D033581:Stem Cell Transplantation; D016212:Transforming Growth Factor beta / Q000037:antagonists & inhibitors / Q000276:immunology
D000305:Adrenal Cortex Hormones; D000617:Aminoglycosides; D000906:Antibodies; D016212:Transforming Growth Factor beta