Molekulare Therapien erblicher Myopathien im Erwachsenenalter – eine kursive Rundschau

Zusammenfassung

Unterschiedliche Formen der molekularen Therapie sind zu einer neuen Möglichkeit in der Präzisionsbehandlung erblicher neuromuskulärer Erkrankungen geworden. Dieser kursive Überblick über die molekularen Therapien bei hereditären Myopathien wird sich auf ausgewählte aktuelle Phase 1 bis 3 Studien zu häufigen hereditären Myopathien im Erwachsenenalter wie die Dystrophinopathie Becker-Kiener, die Fazioskapulohumerale Muskeldystrophie, Calpainopathie, und die Dysferlinopathie fokussieren. Die Therapieoptionen zum Morbus Pompe dienen als Beispiel für die hereditären metabolischen Myopathien.

Abstract

Several types of molecular therapy have become a novel opportunity in the precision treatment of hereditary neuromuscular disorders. This cursive review of gene therapy in hereditary myopathies will focus on selected current phase 1 to 3 trials of common adult hereditary myopathies such as Becker muscular dystrophy, facioscapulohumeral muscular dystrophy, calpainopathy, and dysferlinopathy. The treatment options for Pompe disease serve as an example for hereditary metabolic myopathies.

Schlüsselwörter

Molekulare Therapie - klinische Studien - erbliche Myopathien - Neuromuskuläre Erkrankungen - Überblick

Key words

molecular therapy - clinical trials - hereditary myopathy - neuromuscular disorders - review

Publication History

Received: 28 July 2022

Accepted: 23 September 2022

Article published online:
08 November 2022

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