Cargando…

Systematic Literature Review of Clinical and Economic Evidence for Spinal Muscular Atrophy

INTRODUCTION: The recent advent of disease-modifying therapies (DMTs) has dramatically changed the treatment landscape of spinal muscular atrophy (SMA), and the multifaceted impact of this advancement has not been assessed thoroughly in the growing body of literature. We sought to summarize the lite...

Descripción completa

Detalles Bibliográficos
Autores principales:	Yang, Min, Awano, Hiroyuki, Tanaka, Satoru, Toro, Walter, Zhang, Su, Dabbous, Omar, Igarashi, Ataru
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Springer Healthcare 2022
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9056474/ https://www.ncbi.nlm.nih.gov/pubmed/35307799 http://dx.doi.org/10.1007/s12325-022-02089-2

Ejemplares similares

Economic burden of spinal muscular atrophy: an analysis of claims data
por: Belter, Lisa, et al.
Publicado: (2020)

Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments
por: Dangouloff, Tamara, et al.
Publicado: (2021)

Nusinersen for Spinal Muscular Atrophy in the United States: Findings From a Retrospective Claims Database Analysis
por: Gauthier-Loiselle, Marjolaine, et al.
Publicado: (2021)

Spinal Muscular Atrophy: The Past, Present, and Future of Diagnosis and Treatment
por: Nishio, Hisahide, et al.
Publicado: (2023)

Systematic Literature Review to Assess Economic Evaluations in Spinal Muscular Atrophy (SMA)
por: Paracha, Noman, et al.
Publicado: (2021)

Health Care Resource Utilization and Costs for Patients with Spinal Muscular Atrophy: Findings from a Retrospective US Claims Database Analysis
por: Toro, Walter, et al.
Publicado: (2023)

Correction to: Systematic Literature Review to Assess Economic Evaluations in Spinal Muscular Atrophy (SMA)
por: Paracha, Noman, et al.
Publicado: (2022)

Detection of Spinal Muscular Atrophy Patients Using Dried Saliva Spots
por: Wijaya, Yogik Onky Silvana, et al.
Publicado: (2021)

Spinal muscular atrophy
por: D'Amico, Adele, et al.
Publicado: (2011)

Spinal Muscular Atrophy: Diagnosis, Incidence, and Newborn Screening in Japan
por: Kimizu, Tomokazu, et al.
Publicado: (2021)

Stability and Oligomerization of Mutated SMN Protein Determine Clinical Severity of Spinal Muscular Atrophy
por: Niba, Emma Tabe Eko, et al.
Publicado: (2022)

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 Effectiveness Review (ICER)
por: Dean, Rebecca, et al.
Publicado: (2021)

Nusinersen for spinal muscular atrophy
por: Wurster, Claudia D., et al.
Publicado: (2018)

Spinal Muscular Atrophy and Ependymoma
por: Albakr, Aishah, et al.
Publicado: (2017)

Risdiplam for spinal muscular atrophy
Publicado: (2022)

Disease burden of spinal muscular atrophy in Germany
por: Klug, Constanze, et al.
Publicado: (2016)

Swallowing markers in spinal and bulbar muscular atrophy
por: Banno, Haruhiko, et al.
Publicado: (2017)

Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients
por: Malone, Daniel C., et al.
Publicado: (2019)

Longitudinal characterization of biomarkers for spinal muscular atrophy
por: Bonati, Ulrike, et al.
Publicado: (2017)

High Concentration or Combined Treatment of Antisense Oligonucleotides for Spinal Muscular Atrophy Perturbed SMN2 Splicing in Patient Fibroblasts
por: Wijaya, Yogik Onky Silvana, et al.
Publicado: (2022)

Neuropathology and Therapeutic Intervention in Spinal and Bulbar Muscular Atrophy
por: Banno, Haruhiko, et al.
Publicado: (2009)

Current Status of Treatment of Spinal and Bulbar Muscular Atrophy
por: Tanaka, Fumiaki, et al.
Publicado: (2012)

Modeling Spinal Muscular Atrophy in Drosophila
por: Chang, Howard Chia-Hao, et al.
Publicado: (2008)

Carrier screening for spinal muscular atrophy
por: Prior, Thomas W.
Publicado: (2008)

Androgens in spinal and bulbar muscular atrophy
Publicado: (2012)

Spinal Muscular Atrophy: The Treatment Approved
por: Tabet, Rabih, et al.
Publicado: (2017)

Advances in spinal muscular atrophy therapeutics
por: Parente, Valeria, et al.
Publicado: (2018)

Euglycemic Ketoacidosis in Spinal Muscular Atrophy
por: Stoimenis, Dimitrios, et al.
Publicado: (2019)

Metabolic Dysfunction in Spinal Muscular Atrophy
por: Deguise, Marc-Olivier, et al.
Publicado: (2021)

Update on Biomarkers in Spinal Muscular Atrophy
por: Pino, Megan G, et al.
Publicado: (2021)

Onasemnogene abeparvovec for spinal muscular atrophy
Publicado: (2022)

Mitochondrial Dysfunction in Spinal Muscular Atrophy
por: Zilio, Eleonora, et al.
Publicado: (2022)

Editorial: Advances in spinal muscular atrophy
por: Sun, Jianli
Publicado: (2023)

Spinal muscular atrophy disease: a literature review for therapeutic strategies
por: Stavarachi, M, et al.
Publicado: (2010)

A National Spinal Muscular Atrophy Registry for Real-World Evidence
por: Hodgkinson, Victoria L., et al.
Publicado: (2020)

PCR-Based Screening of Spinal Muscular Atrophy for Newborn Infants in Hyogo Prefecture, Japan
por: Noguchi, Yoriko, et al.
Publicado: (2022)

Limited Phenotypic Effects of Selectively Augmenting the SMN Protein in the Neurons of a Mouse Model of Severe Spinal Muscular Atrophy
por: Lee, Andrew J-H., et al.
Publicado: (2012)

Survival analysis of spinal muscular atrophy type I
por: Park, Hyun Bin, et al.
Publicado: (2010)

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy
por: Al‐Zaidy, Samiah, et al.
Publicado: (2018)

Observation of the natural course of type 3 spinal muscular atrophy: data from the polish registry of spinal muscular atrophy
por: Lusakowska, Anna, et al.
Publicado: (2021)

Cannot write session to /tmp/vufind_sessions/sess_gavupse8if9jsd2gorofdacmjq