Home > Neurology > AAN 2022 > Muscle and Neuro-Muscular Disorders > Losmapimod for facioscapulohumeral muscular dystrophy

Losmapimod for facioscapulohumeral muscular dystrophy

Presented By
Prof. Al-Rabi N. Tawil, University of Rochester, NY, USA
Presented by
Al-Rabi Tawil University of Rochester
AAN 2022
Losmapimod showed improvement on relevant clinical endpoints in the treatment of facioscapulohumeral muscular dystrophy (FSHD) in the ReDUX4 trial. Its favourable safety and tolerability results support continued development. FSHD is caused by the misexpression of the double homeobox protein 4 (DUX4) transcription factor in skeletal muscle. This condition ultimately results in progressive motor disability. Currently, no treatment options are available for FSHD that prevent or slow muscle weakness and wasting. Losmapimod is an orally active, selective, small molecule inhibitor of p38α/β that reduced DUX4. It has been evaluated in over 3,500 subjects in clinical trials across 10 other indications, with no safety signals. The efficacy and safety results of losmapimod from the phase 2 ReDUX4 trial were presented by Prof. Al-Rabi N. Tawil (University of Rochester, NY, USA) [1]. Enrolled were 80 FSHD participants aged 18–65 years (mean age 46 years) wh...

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