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Ongoing clinical trials for DMD/BMD and LGMD diseases. The current stage at the time of writing for the most advanced in each category is illustrated. Indicatively “Exon skipping” drugs is a promising class, in which Drisapersen/PRO051 (Prosensa), Eteplirsen/AVI-4658 (Sarepta) are currently in phase III [65] Ataluren/PTC-124 has received marketing approval in Europe (but not in US) under the name Translarna mda.gov.
