Refining Patient Pool Selection to Overcome Patient Heterogeneity Challenges & Improve Clinical Success
3:15 pm - Thursday 28th January 2027
- Leveraging genetic, molecular and disease-specific biomarkers to distinguish patient subgroups within Duchenne, Becker, FSHD, DM1 and LGMD and strengthen the interpretation of clinical outcomes
- Characterizing differences in ambulatory status, muscle degeneration patterns and rates of disease progression across patients
