Prevalence of morbidities across the lifespan for adults with spinal muscular atrophy: a retrospective cohort study

por G. Whitney, Daniel, E. Neil Knierbein, Erin, K. Daunter, Alecia
Publicado 2023

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial

por Finkel, Richard S., McDonald, Craig M., Lee Sweeney, H., Finanger, Erika, Neil Knierbein, Erin, Wagner, Kathryn R., Mathews, Katherine D., Marks, Warren, Statland, Jeffrey, Nance, Jessica, McMillan, Hugh J., McCullagh, Gary, Tian, Cuixia, Ryan, Monique M., O’Rourke, Declan, Müller-Felber, Wolfgang, Tulinius, Mar, Burnette, W. Bryan, Nguyen, Cam-Tu, Vijayakumar, Kayal, Johannsen, Jessika, Phan, Han C., Eagle, Michelle, MacDougall, James, Mancini, Maria, Donovan, Joanne M.
Publicado 2021