Pharmacologic and genetic therapy for childhood muscular dystrophies.
The outstanding advances in the molecular characterization of muscle diseases, including muscular dystrophies, inflammatory myopathies, and ion channel disorders, have resulted in the identification of potential targets for pharmacologic and genetic therapy in the best characterized of these diseases. The most common myopathy in children, Duchenne muscular dystrophy (DMD), is the focus of active pharmacologic clinical trials. Genetic transfer therapy research for this and other dystrophies is rapidly moving forward. However, as new approaches for treatment are being actively investigated, the current modality of treatment for all myopathies is still in the realm of physical medicine and rehabilitation. The focus of this review is on the advances in pharmacologic and genetic therapy research in DMD and limb girdle muscular dystrophies.
Escolar
D M
DM
Department of Neurology, Research Center for Genetic Medicine, MDA Clinic, Children's National Medical Center, George Washington University, 111 Michigan Avenue NW, Washington, DC 20010, USA. descolar@childrens-research.org
Scacheri
C G
CG
D000818:Animals; D017690:Cell Transplantation; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic; D003401:Creatine / Q000627:therapeutic use; D003598:Cytoskeletal Proteins / Q000235:genetics / Q000502:physiology; D004334:Drug Administration Schedule; D016189:Dystrophin / Q000235:genetics; D005260:Female; D005786:Gene Expression Regulation; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000627:therapeutic use; D006801:Humans; D008297:Male; D008565:Membrane Proteins / Q000235:genetics / Q000502:physiology; D051379:Mice; D018101:Mice, Inbred mdx; D018482:Muscle, Skeletal / Q000166:cytology; D009136:Muscular Dystrophies / Q000188:drug therapy / Q000503:physiopathology / Q000628:therapy*; D009137:Muscular Dystrophy, Animal / Q000628:therapy; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy / Q000503:physiopathology / Q000628:therapy; D011241:Prednisone / Q000009:adverse effects / Q000627:therapeutic use; D011282:Pregnenediones / Q000627:therapeutic use; D014176:Protein Biosynthesis; D012508:Sarcolemma / Q000473:pathology; D049411:Utrophin
D003598:Cytoskeletal Proteins; D016189:Dystrophin; D008565:Membrane Proteins; D011282:Pregnenediones; C483600:Utrn protein, mouse; D049411:Utrophin; C021988:deflazacort; D003401:Creatine; D011241:Prednisone