Efficacy and security of onasemnogene abeparvovec-XIOI in spinal muscular atrophy: a systematic review and meta-analysis

2021 ◽  
Vol 429 ◽  
pp. 118887
Author(s):  
Aaron Rodriguez-Calienes ◽  
Sandra Chavez-Malpartida ◽  
Giancarlo Alvarado-Gamarra ◽  
Niels Pacheco-Barrios ◽  
Peggy Martinez-Esteban ◽  
...  
Keyword(s):  
Systematic Review ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Meta Analysis

Efficacy and Safety of Valproic Acid for Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis

CNS Drugs ◽  
2019 ◽  
Vol 33 (3) ◽  
pp. 239-250
Author(s):  
Abdelrahman Elshafay ◽  
Truong Hong Hieu ◽  
Mohamed Fahmy Doheim ◽  
Mahmoud Attia Mohamed Kassem ◽  
Mohammed Fathi ELdoadoa ◽  
...  
Keyword(s):  
Systematic Review ◽  
Valproic Acid ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Meta Analysis ◽  
Efficacy And Safety

scholarly journals Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models

Gene Therapy ◽  
2021 ◽  
Author(s):  
Ellie M. Chilcott ◽  
Evalyne W. Muiruri ◽  
Theodore C. Hirst ◽  
Rafael J. Yáñez-Muñoz
Keyword(s):  
Systematic Review ◽  
Experimental Design ◽  
Spinal Muscular Atrophy ◽  
Publication Bias ◽  
Median Survival ◽  
Muscular Atrophy ◽  
Meta Analysis ◽  
Significant Heterogeneity ◽  
Rodent Models ◽  
Genetic Therapy

AbstractSpinal muscular atrophy (SMA) is a severe childhood neuromuscular disease for which two genetic therapies, Nusinersen (Spinraza, an antisense oligonucleotide), and AVXS-101 (Zolgensma, an adeno-associated viral vector of serotype 9 AAV9), have recently been approved. We investigated the pre-clinical development of SMA genetic therapies in rodent models and whether this can predict clinical efficacy. We have performed a systematic review of relevant publications and extracted median survival and details of experimental design. A random effects meta-analysis was used to estimate and compare efficacy. We stratified by experimental design (type of genetic therapy, mouse model, route and time of administration) and sought any evidence of publication bias. 51 publications were identified containing 155 individual comparisons, comprising 2573 animals in total. Genetic therapies prolonged survival in SMA mouse models by 3.23-fold (95% CI 2.75–3.79) compared to controls. Study design characteristics accounted for significant heterogeneity between studies and greatly affected observed median survival ratios. Some evidence of publication bias was found. These data are consistent with the extended average lifespan of Spinraza- and Zolgensma-treated children in the clinic. Together, these results support that SMA has been particularly amenable to genetic therapy approaches and highlight SMA as a trailblazer for therapeutic development.

Biomarkers of disease progression in adolescents and adults with 5q spinal muscular atrophy: a systematic review and meta-analysis

2022 ◽  
Author(s):  
Maria Gavriilaki ◽  
Maria Moschou ◽  
Vasileios Papaliagkas ◽  
Konstantinos Notas ◽  
Evangelia Chatzikyriakou ◽  
...  
Keyword(s):  
Systematic Review ◽  
Spinal Muscular Atrophy ◽  
Disease Progression ◽  
Muscular Atrophy ◽  
Meta Analysis ◽  
Adolescents And Adults

scholarly journals Systematic review of motor function scales and patient reported outcomes in spinal muscular atrophy

2021 ◽  
Vol Publish Ahead of Print ◽  
Author(s):  
Jennifer W. Wu ◽  
Laura Pepler ◽  
Bridget Maturi ◽  
Alexandria C. F. Afonso ◽  
Janice Sarmiento ◽  
...  
Keyword(s):  
Systematic Review ◽  
Spinal Muscular Atrophy ◽  
Motor Function ◽  
Patient Reported Outcomes ◽  
Muscular Atrophy ◽  
Patient Reported

scholarly journals Functional Rating Scales in Spinal and Bulbar Muscular Atrophy: A Systematic Review, Meta-Analysis and Critical Appraisal of their Measurement Properties

2021 ◽  
Author(s):  
Agessandro Abrahao ◽  
LIANE PHUNG ◽  
Eliza Freitas ◽  
Cornelia M Borkhoff ◽  
Lorne Zinman
Keyword(s):  
Systematic Review ◽  
Motor Function ◽  
Rating Scales ◽  
Rating Scale ◽  
Muscular Atrophy ◽  
Meta Analysis ◽  
Motor Dysfunction ◽  
Measurement Properties ◽  
Patient Reported ◽  
Spinal Bulbar Muscular Atrophy

Tracking disease progression and treatment effect of spinal bulbar muscular atrophy, or Kennedy's disease, is challenging given its slowly progressive nature. To achieve success in SBMA clinical trials, a reliable, responsive, and validated patient-reported motor function scale must capture progression of SBMA-specific motor dysfunction. Here, we conducted a systematic review, meta-analysis, and appraisal of core measurement properties of the SBMA functional rating scale (SBMAFRS). We established that the SBMAFRS has satisfactory internal consistency, inter-rater reliability, and construct validity for measuring progressive motor dysfunction over similar neurodegenerative motor function scales but inadequate sensitivity to change over time. Further development to validate and improve the SBMAFRS' ability to capture longitudinal responsiveness in larger cohorts is warranted.

Quality of life of patients with spinal muscular atrophy: A systematic review

2019 ◽  
Vol 23 (3) ◽  
pp. 347-356 ◽  
Author(s):  
Erik Landfeldt ◽  
Josefin Edström ◽  
Thomas Sejersen ◽  
Már Tulinius ◽  
Hanns Lochmüller ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Systematic Review ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy

Describing nutrition in spinal muscular atrophy: A systematic review

2016 ◽  
Vol 26 (7) ◽  
pp. 395-404 ◽  
Author(s):  
Georgia E. Moore ◽  
Amara W. Lindenmayer ◽  
Grace A. McConchie ◽  
Monique M. Ryan ◽  
Zoe E. Davidson
Keyword(s):  
Systematic Review ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy

PP09.1 – 2352: Systematic review of incidence and prevalence of spinal muscular atrophy (SMA)

2015 ◽  
Vol 19 ◽  
pp. S64-S65 ◽  
Author(s):  
C. Jones ◽  
M. Oskoui ◽  
D. Zielinski ◽  
L. Vinikoor ◽  
W. Farwell
Keyword(s):  
Systematic Review ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy
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