A prognostic model for predicting individual 5-year motor function trajectories in Duchenne muscular dystrophy patients using the North Star Ambulatory Assessment.
Establishes minimal detectable change thresholds for the North Star Ambulatory Assessment in Duchenne muscular dystrophy, accounting for variation across the functional spectrum.
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.
Machine learning predictive model for quality of life deterioration after risankizumab withdrawal in psoriasis, identifying patient factors that inform re-treatment decisions.
This study evaluates Maryland's global budget reform in rural hospitals, finding that the reform decreased outpatient utilization but did not significantly affect inpatient admissions.
Using Health and Retirement Study data linked to occupational classifications, this study examines how lifetime job demands relate to disability in later life.
Using data from the VHA linked to Medicare claims, this study analyzed the rate to which Veterans at high risk of stroke received recommended anticoagulation therapy, finding suboptimal rates of guideline-concordant prescribing.