Facioscapulohumeral muscular dystrophy: the road to targeted therapies

Nature Reviews Neurology, Published online: 10 January 2023; doi:10.1038/s41582-022-00762-2Facioscapulohumeral muscular dystrophy is caused by aberrant expression of the transcription factor DUX4. Tihaya, Mul and colleagues describe advances in the development of targeted treatments for facioscapulohumeral muscular dystrophy and discuss potential clinical trial outcome measures as well as molecular and imaging biomarkers.

Source: Nature Reviews Neurology - January 10, 2023 Category: Neurology Authors: Mara S. Tihaya Karlien Mul Judit Balog Jessica C. de Greef Stephen J. Tapscott Rabi Tawil Jeffrey M. Statland Silv ère M. van der Maarel Source Type: research