High Healthcare Resource Use in Hospitalized Patients with a Diagnosis of Spinal Muscular Atrophy Type 1 (SMA1): Retrospective Analysis of the Kids’ Inpatient Database (KID)

por Cardenas, Jessica, Menier, Melissa, Heitzer, Marjet D., Sproule, Douglas M.
Publicado 2018

Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients

por Malone, Daniel C., Dean, Rebecca, Arjunji, Ramesh, Jensen, Ivar, Cyr, Phil, Miller, Beckley, Maru, Benit, Sproule, Douglas M., Feltner, Douglas E., Dabbous, Omar
Publicado 2019

Response to: Alfred Sandrock, Wildon Farwell. Letter to the Editor, Comparisons Between Separately Conducted Clinical Trials: Letter to the Editor Regarding Dabbous O, Maru B, Jans...

por Dabbous, Omar, Maru, Benit, Jansen, Jeroen P., Lorenzi, Maria, Cloutier, Martin, Guérin, Annie, Pivneva, Irina, Wu, Eric Q., Arjunji, Ramesh, Feltner, Douglas, Sproule, Douglas M.
Publicado 2019

Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1

por Dabbous, Omar, Maru, Benit, Jansen, Jeroen P., Lorenzi, Maria, Cloutier, Martin, Guérin, Annie, Pivneva, Irina, Wu, Eric Q., Arjunji, Ramesh, Feltner, Douglas, Sproule, Douglas M.
Publicado 2019

An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and...

por Dean, Rebecca, Jensen, Ivar, Cyr, Phil, Miller, Beckley, Maru, Benit, Sproule, Douglas M., Feltner, Douglas E., Wiesner, Thomas, Malone, Daniel C., Bischof, Matthias, Toro, Walter, Dabbous, Omar
Publicado 2021

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

por Al‐Zaidy, Samiah, Pickard, A. Simon, Kotha, Kavitha, Alfano, Lindsay N., Lowes, Linda, Paul, Grace, Church, Kathleen, Lehman, Kelly, Sproule, Douglas M., Dabbous, Omar, Maru, Benit, Berry, Katherine, Arnold, W. David, Kissel, John T., Mendell, Jerry R., Shell, Richard
Publicado 2018

SMA-MAP: A Plasma Protein Panel for Spinal Muscular Atrophy

por Kobayashi, Dione T., Shi, Jing, Stephen, Laurie, Ballard, Karri L., Dewey, Ruth, Mapes, James, Chung, Brett, McCarthy, Kathleen, Swoboda, Kathryn J., Crawford, Thomas O., Li, Rebecca, Plasterer, Thomas, Joyce, Cynthia, Chung, Wendy K., Kaufmann, Petra, Darras, Basil T., Finkel, Richard S., Sproule, Douglas M., Martens, William B., McDermott, Michael P., De Vivo, Darryl C., Walker, Michael G., Chen, Karen S.
Publicado 2013