New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges

by Sonia Messina, Maria Sframeli
Published 2020-07-01

Spinal muscular atrophy - onasemnogene abeparvovec and other therapeutic options

by Aleksandra Alicja Majchrzak-Celińska, Anna Warych, Mikołaj Szoszkiewicz
Published 2020-02-01

2020 Update to Spinal Muscular Atrophy Management in Saudi Arabia

by Fahad A. Bashiri, Fahad A. Bashiri, Mohamad-Hani Temsah, Mohamad-Hani Temsah, Khalid Hundallah, Fahad Alsohime, Fahad Alsohime, Yazed AlRuthia, Yazed AlRuthia
Published 2021-05-01

INNOVATIVE THERAPIES IN GENETIC DISEASES: SPINAL MUSCULAR ATROPHY

by Elena-Silvia Shelby, Andrada Mirea
Published 2021-06-01

Review of therapeutic options for spinal muscular atrophy

by Singh Arun, Jain Monica, Kapadia Rupa, Mahawar-Dhirendra Kumar, Kakkar Shivankan, Dadhich Jaya, Chandel-Ritesh Kumar
Published 2021-01-01

Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment

by Alisha Monnette, Er Chen, Dongzhe Hong, Alessandra Bazzano, Stacy Dixon, W. David Arnold, Lizheng Shi
Published 2021-01-01

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...

by Rebecca Dean, Ivar Jensen, Phil Cyr, Beckley Miller, Benit Maru, Douglas M. Sproule, Douglas E. Feltner, Thomas Wiesner, Daniel C. Malone, Matthias Bischof, Walter Toro, Omar Dabbous
Published 2021-01-01

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

by Daniel C. Malone, Rebecca Dean, Ramesh Arjunji, Ivar Jensen, Phil Cyr, Beckley Miller, Benit Maru, Douglas M. Sproule, Douglas E. Feltner, Omar Dabbous
Published 2019-01-01