Results 191 to 200 of about 3,107 (205)

An updated systematic review on spinal muscular atrophy patients treated with nusinersen, onasemnogene abeparvovec (at least 24 months), risdiplam (at least 12 months) or combination therapies.

European journal of paediatric neurology
Doris Giess, Judit Erdos, Claudia Wild
semanticscholar   +1 more source

415PIntrathecal onasemnogene abeparvovec for patients with spinal muscular atrophy: phase 3, randomized, sham-controlled, double-blind STEER study

Neuromuscular Disorders
C. Proud, D. Vũ, J. Wilmshurst, O. Sanmaneechai, S. Gulati, H. Xiong, H. C. Moreno, S. Tay, M. Thong, A. Born, A. B. Ortega, Y. Jong, M. Al-muhaizea, A. Lee, J. Visootsak, S. Tauscher-Wisniewski, I. Alecu, R. Parlikar, R. Finkel   +18 more
semanticscholar   +1 more source

AEs of nusinersen, risdiplam and onasemnogene abeparvovec

Reactions Weekly, 2023
openaire   +1 more source

518PClinical outcome and effectiveness of onasemnogene abeparvovec in treatment-naïve spinal muscular atrophy

Neuromuscular Disorders
H. Lee
semanticscholar   +1 more source

583PAdd-on treatment with risdiplam after treatment with onasemnogene abeparvovec: case report of a 2.5-year-old patient with SMA type 1

Neuromuscular Disorders
M. Drax, M. Pühringer, A. Eisenkölbl, G. Gröppel   +3 more
semanticscholar   +1 more source

Real-world experience with switch to onasemnogene abeparvovec after initial therapy with nusinersen or risdiplam

Neuromuscular Disorders
Magdalena Chrościńska-Krawczyk, Ilona Kozioł, Ewa Zienkiewicz   +2 more
semanticscholar   +1 more source

Treatment Outcome of Risdiplam and Onasemnogene Abeparvovec in an Infant With Spinal Muscular Atrophy Dependent on Prolonged Invasive Ventilation

Journal of Paediatrics and Child Health
Jia Wei Audrey Lee, Huixin Huang, F. Wang, S. K. Tay   +3 more
semanticscholar   +1 more source

Onasemnogene abeparvovec (Zolgensma)

2023
openaire   +1 more source

Onasemnogene abeparvovec-related AEs in infants with SMA

Reactions weekly

semanticscholar   +1 more source

Safety of onasemnogene abeparvovec in an infant with spinal muscular atrophy type 1 and severe neutropenia: a case report and literature review

Medical Reports
Maria Moutafi, Nikolaos Gkiourtzis, Maria Papadopoulou, Efterpi Dalpa, Athanassios Evangeliou, P. Panagopoulou   +5 more
semanticscholar   +1 more source