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Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study

De Vivo, D. C.; Bertini, E.; Swoboda, K. J.; Hwu, W. L.; Crawford, T. O.; Finkel, R. S.; Kirschner, J.; Kuntz, N. L.; Parsons, J. A.; Ryan, M. M.; Butterfield, R. J.; Topaloglu, H.; Ben-Omran, T.; Sansone, V. A.; Jong, Y. J.; Shu, F.; Staropoli, J. F.; Kerr, D.; Sandrock, A. W.; Stebbins, C.; Petrillo, M.; Braley, G.; Johnson, K.; Foster, R.; Gheuens, S.; Bhan, I.; Reyna, S. P.; Fradette, S.; Farwell, W. Neuromuscul Disord. 2019 Nov 11; 29(11):842-856

Neurodevelopmental Outcomes Among Children With Congenital Heart Disease: At-Risk Populations and Modifiable Risk Factors

Ryan, K. R.; Jones, M. B.; Allen, K. Y.; Marino, B. S.; Casey, F.; Wernovsky, G.; Lisanti, A. J. World J Pediatr Congenit Heart Surg. 2019 Oct 30; 10(6):750-758

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function

Hoffman, E. P.; Schwartz, B. D.; Mengle-Gaw, L. J.; Smith, E. C.; Castro, D.; Mah, J. K.; McDonald, C. M.; Kuntz, N. L.; Finkel, R. S.; Guglieri, M.; Bushby, K.; Tulinius, M.; Nevo, Y.; Ryan, M. M.; Webster, R.; Smith, A. L.; Morgenroth, L. P.; Arrieta, A.; Shimony, M.; Siener, C.; Jaros, M.; Shale, P.; McCall, J. M.; Nagaraju, K.; van den Anker, J.; Conklin, L. S.; Cnaan, A.; Gordish-Dressman, H.; Damsker, J. M.; Clemens, P. R. Neurology. 2019 Aug 28; 93(13):e1312-e1323

Prohibitin is a prognostic marker and therapeutic target to block chemotherapy resistance in Wilms' tumor

Ortiz, M. V.; Ahmed, S.; Burns, M.; Henssen, A. G.; Hollmann, T. J.; MacArthur, I.; Gunasekera, S.; Gaewsky, L.; Bradwin, G.; Ryan, J.; Letai, A.; He, Y.; Naranjo, A.; Chi, Y. Y.; LaQuaglia, M.; Heaton, T.; Cifani, P.; Dome, J. S.; Gadd, S.; Perlman, E.; Mullen, E.; Steen, H.; Kentsis, A. JCI Insight. 2019 Aug 9; 4(15)