From our webinar on November 7, 2018. Dr. Tapscott, one of the world’s leading scientists working on the fundamental understanding of FSH muscular dystrophy, updates us on our understanding of the condition. Magnetic resonance imaging (MRI) data suggest that muscle degeneration begins with local inflammation followed by degenerative changes (“fatty infiltration”) in a muscle. Tapscott discusses a recently published study that correlated changes seen in MRI with evidence of muscle damage and activation of the DUX4 gene, which is widely thought to trigger the disease process. Findings like these provide the knowledge base for designing clinical trials to test DUX4-suppressing drugs in patients.
Posted on: Nov 16, 2018