Rabi Tawil, Kathryn R Wagner, Johanna I Hamel, Doris G Leung, Jeffrey M Statland, Leo H Wang, Angela Genge, Sabrina Sacconi, Hanns Lochmüller, David Reyes-Leiva, Jordi Diaz-Manera, Jorge Alonso-Perez, Nuria Muelas, Juan J Vilchez, Alan Pestronk, Summer Gibson, Namita A Goyal, Lawrence J Hayward, Nicholas Johnson, Samantha LoRusso, Miriam Freimer, Perry B Shieh, S H Subramony, Baziel van Engelen, Joost Kools, Olof Dahlqvist Leinhard, Per Widholm, Christopher Morabito, Christopher M Moxham, Diego Cadavid, Michelle L Mellion, Adefowope Odueyungbo, William G Tracewell, Anthony Accorsi, Lucienne Ronco, Robert J Gould, Jennifer Shoskes, Luis Alejandro Rojas, John G Jiang
BACKGROUND: Facioscapulohumeral muscular dystrophy is a hereditary progressive myopathy caused by aberrant expression of the transcription factor DUX4 in skeletal muscle. No approved disease-modifying treatments are available for this disorder. We aimed to assess the safety and efficacy of losmapimod (a small molecule that inhibits p38α MAPK, a regulator of DUX4 expression, and p38β MAPK) for the treatment of facioscapulohumeral muscular dystrophy. METHODS: We did a randomised, double-blind, placebo-controlled phase 2b trial at 17 neurology centres in Canada, France, Spain, and the USA...
May 2024: Lancet Neurology