Molecular Therapies of Hereditary Myopathies in Adulthood - a Cursive Overview

Schoser, Benedikt (2022): Molecular Therapies of Hereditary Myopathies in Adulthood - a Cursive Overview. In: Fortschritte der Neurologie - Psychiatrie, Bd. 91, Nr. 4: S. 164-168

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.

• Dokumententyp: Zeitschriftenartikel
• Fakultät: Medizin
• Themengebiete: 600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin und Gesundheit
• ISSN: 0720-4299
• Sprache: Englisch