A prognostic model for predicting individual 5-year motor function trajectories in Duchenne muscular dystrophy patients using the North Star Ambulatory Assessment.
Long-term survival analysis comparing eteplirsen-treated Duchenne muscular dystrophy patients to matched natural history controls, demonstrating survival benefits with treatment.
A framework for developing and validating multi-domain composite endpoints for AL amyloidosis clinical trials, developed through a public-private partnership with the FDA.
Long-term analysis of health-related quality of life outcomes in hereditary transthyretin amyloidosis patients treated with inotersen, demonstrating sustained benefits over extended follow-up.