[Therapy for myopathies: from management to genetic treatment: introductory remarks].
Despite a wealth of recent information about the molecular basis of Duchenne muscular dystrophy (DMD), there is no effective treatment for this condition because the mechanism how dystrophin deficiency produces the muscle fiber degradation is unknown. Much effort has been made to develop a new treatment for DMD on clinical and molecular bases. This symposium consist of reports on recent advance of the gene, molecular and clinical therapies for DMD. It also discusses recent topics of therapeutic trial for glycogen storage diseases, featuring recombinant human acid alpha-glucosidase GAA enzyme therapy for fatal glycogen storage disease type II.
Miike
Teruhisa
T
Department of Child Development, Faculty of Medical and Pharmaceutical Sciences, Kumamoto University Graduate School, Kumamoto. miketeru@kaiju.medic.kumamoto-u.ac.jp
Osawa
Makiko
M
D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D009135:Muscular Diseases / Q000209:etiology / Q000628:therapy*