Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: A systematic review of real-world study data [PDF]
, 2022 Judit Erdös, Claudia Wild
openalex +1 more source
“Not Only Has She Survived, But She Lives a Happy Life”: Parents’ Perspectives and Experiences of a Novel Disease-Modifying Therapy for Spinal Muscular Atrophy in Sweden [PDF]
The objective of this prospective cohort study was to explore parents' perspectives of patients' experiences of the first available novel disease-modifying therapy for SMA in Sweden.
Cortina-Borja, Mario +4 more
core
Safety, Tolerability, and Effect of Nusinersen Treatment in Ambulatory Adults With 5q-SMA
, 2021 Bakri Elsheikh +14 more
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Muscle MRI in two SMA patients on nusinersen treatment: A two years follow-up [PDF]
, 2020 Andrea Barp +11 more
openalex +1 more source
A historical and current review of newborn screening for neuromuscular disorders from around the world: lessons for the United States [PDF]
, 2017 Clarke, Angus John +1 more
core +2 more sources
The Antisense Oligonucleotide Nusinersen for Treatment of Spinal Muscular Atrophy [PDF]
, 2021 Amber N. Edinoff +8 more
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Safety and Efficacy of Nusinersen Focusing on Renal and Hematological Parameters in Spinal Muscular Atrophy [PDF]
Hüseyin Bahadır Şenol +6 more
openalex +1 more source
Cost comparison analysis of onasemnogene abeparvovec and nusinersen for treatment of patients with spinal muscular atrophy type 1 in the Netherlands [PDF]
Simon van der Schans +7 more
openalex +1 more source
Nusinersen versus sham control in later-onset spinal muscular atrophy [PDF]
, 2018 Connolly, A. M., et al,
core +1 more source
Matched-pair analysis of motor outcomes in adults with spinal muscular atrophy on nusinersen vs. risdiplam. [PDF]
J NeurolSvenja N +11 more
europepmc +1 more source