Treating Muscle Atrophy in FSHD Patients

SLU ID 17-017 | p38 Inhibitors To Reduce DUX4 Expression For Treatment of FSHD

Background

Facioscapulohumeral Muscular Dystrophy (FSHD) is characterized by epigenetic changes resulting in the aberrant expression of the DUX4 gene in muscle. The Toxic DUX4 protein causes muscle degeneration through many potential mechanisms including induction of muscle cell death and inhibition of muscle regeneration. Suppression of DUX4 expression is therefore considered a primary therapeutic approach for halting disease progression in FSHS; however, the mechanisms responsible for DUX expression are poorly understood and few drug targets have been described. p38 inhibitors suppresses toxic DUX4 expression in FSHD muscle cells and therefore provides disease modification by halting the long term degenerative process that result in atrophy of muscle and loss of function in FSHD patients.

Overview

Researchers at Saint Louis University have developed a method utilizing p38 inhibitors to suppress DUX4 expression FSHD patients to halt muscle atrophy.

Benefits

The potential benefits of this technology include:

• Minimizing muscle degeneration

• Minimizing disease progression

Applications

The potential applications of this technology include treatments for FSHD.

Opportunity

Saint Louis University is seeking partners to further develop and commercialize this technology.