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James Signorovitch

Predicting North Star Ambulatory Assessment trajectories in Duchenne muscular dystrophy: A mixed-effects modeling approach
Minimal detectable change and meaningful change in motor function in Duchenne muscular dystrophy
Pathway for Development and Validation of Multi-domain Endpoints for Amyloid Light Chain (AL) Amyloidosis
Patient characteristics associated with all-cause healthcare costs of alopecia areata in the United States
Long-term treatment effects of inotersen on health-related quality of life in patients with hATTR amyloidosis with polyneuropathy: Analysis of the open-label extension of the NEURO-TTR trial