Article Highlights:
- A multicenter research team has provided specific evidence that inappropriate production of DUX4 in muscle is a major contributor to FSH dystrophy.
- DUX4 disrupts numerous biochemical pathways in muscle, interfering with the ability of muscle cells to develop and thrive.
- Interfering with DUX4 is a promising strategy for the treatment of FSHD.
- Measuring DUX4-related biochemical changes in the body could provide researchers with new biomarkers with which to follow disease progression and response to treatment.
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