Sverdrup Lab Suppresses Toxic Protein in FSHD

Fran Sverdrup, Ph.D., and his research group have identified a potential new drug therapy for the treatment of facioscapulohumeral muscualr dystrophy (FSHD). Their findings were published in the Journal of Pharmacology and Experimental Therapeutics.

FSHD is an inherited disease affecting mainly young adults. It is caused by mis-expression of the double homeobox 4 (DUX4) transcription factor in skeletal muscle, which is silenced in normal skeletal muscle. Sverdrup and colleagues found that an existing drug, a p38 inhibitor, supressed DUX4 expression in an animal model of FSHD as well as in FSHD cells.

In addition to these findings, a pharamceutical company has started a clinical trial of the p38 inhibitor, losmapimod, in patients to test the efficacy in human subjects. These latest findings bring the possibility of a treatment for FSHD that much closer to the affected patients.

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Department of Biochemistry and Molecular Biology