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Hintergrund Neuromuskuläre Erkrankungen galten lange Zeit als nur symptomatisch behandelbar. Durch neue Technologien konnten in den letzten Jahren erstmals kausale Therapien entwickelt werden. Gentherapien für die spinale Muskelatrophie, die Duchenne-Muskeldystrophie, die Gliedergürteldystrophien, die myotubuläre Myopathie und die hereditäre motorisch-sensible Neuropathie Typ 1A werden aktuell in...
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