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Aktuelle Neurologie 2006; 33(6): 328-341
DOI: 10.1055/s-2005-915455
Übersicht
© Georg Thieme Verlag KG Stuttgart · New York

Therapie der Muskeldystrophien

Treatment of Muscular DystrophiesJ.  A.  Petersen1 , H.  Lochmüller1 , M.  C.  Walter1
  • 1Friedrich-Baur-Institut und Labor für Molekulare Myologie an der Neurologischen Klinik und Poliklinik, LMU München
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Publikationsverlauf

Publikationsdatum:
14. März 2006 (online)

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Zusammenfassung

Muskeldystrophien (MD) sind eine heterogene Gruppe genetisch determinierter, progredienter Erkrankungen der Muskulatur. Gemeinsames Merkmal aller Muskeldystrophien ist eine fortschreitende Muskelschwäche und -atrophie, deren Schweregrad und Verteilungsmuster bei den verschiedenen MD-Formen allerdings deutliche Unterschiede zeigt. Infolge der Entdeckung immer neuer Genorte, spezifischer Gene und Genprodukte erfolgt die Klassifizierung der Muskeldystrophien nicht mehr allein anhand klinischer Charakteristika, sondern entsprechend den zugrunde liegenden Gendefekten. In vielen Fällen ermöglich die exakte Diagnose Aussagen zu Verlauf und Prognose der Erkrankung. Leider stehen bislang meist nur symptomatische und supportive Therapien zur Verfügung, darunter pharmakologische, physiotherapeutische und chirurgische. Viel versprechende kausale Behandlungsstrategien wie Gen- und Zelltherapie werden zurzeit entwickelt und teils mit Erfolg am Tiermodell erprobt. Diese Übersicht fasst etablierte Therapieoptionen sowie die Perspektiven molekularmedizinischer Ansätze zusammen.

Abstract

Muscular dystrophies (MD) are a clinically and genetically heterogeneous group of skeletal muscle diseases. Their common features are progressive muscle wasting and atrophy of varying degree. Since the discovery of new gene loci, genes and gene products continues, MDs are no longer classified by their clinical features alone, but by the underlying gene defects. In many cases, exact diagnosis enables predictions concerning course and prognosis of the disease. Therapy is based on symptomatic and supportive approaches, amongst them drugs, physiotherapy and surgery. Promising causal treatment strategies like gene and cell therapy are currently being developed and some of them are succesfully applied in animal models. This review summarizes established therapeutical options as well as the perspectives of molecular medicine in MD.

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Priv.-Doz. Dr. Maggie C. Walter

Friedrich-Baur-Institut und Labor für Molekulare Myologie an der Neurologischen Klinik und Poliklinik · Ludwig-Maximilians-Universität München

Ziemssenstraße 1a

80336 München

eMail: maggie.walter@lrz.uni-muenchen.de

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