Halting and Reversing the Progression of Facioscapulohumeral Muscular Dystrophy (FSHD)

SLU ID 18-023 | Pyrrolopyridinones

Background

Facioscapulohumeral Muscular Dystrophy (FSHD) is a rare but devastating disease that affects 1 in 20,000 individuals. Current research indicates that FSHD is caused by the mis-expression of teh DUX4 gene in muscle, which leads to muscle cell death.

Overview

Researchers at Saint Louis University have discovered a series of pyrrolopyridinone compounds that inhibit BET bromodomains. The compounds also repress the expression of DUX4 and its downstream gene targets in FSHD patient-derived muscle cells.

Benefits

The potential benefits of this technology include:

• Minimizing the progression of FSHD

• Increasing the ability to halt the progression of FSHD.

• Increasing the ability to reverse the progrression of FSHD

• Increasing quality of life for FSHD patients

• Minimizing the symptoms of FSHD

Applications

Potential applications of this technology include developing treatments for Facioscapulohumeral Muscular Dystrophy (FSHD).

Opportunity

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