Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study-Reference-Cited by-同舟云学术

Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study

Published:2021-02-16 Issue:5 Volume:63 Page:668-677
ISSN:0148-639X
Container-title:Muscle & Nerve
language:en
Short-container-title:Muscle Nerve

Author:

Acsadi Gyula¹,Crawford Thomas O.²^ORCID,Müller‐Felber Wolfgang³,Shieh Perry B.⁴^ORCID,Richardson Randal⁵,Natarajan Niranjana⁶,Castro Diana⁷,Ramirez‐Schrempp Daniela⁸,Gambino Giulia⁹,Sun Peng⁸,Farwell Wildon⁸

Affiliation:

1. Division of Pediatric Neurology, Connecticut Children's Medical Center University of Connecticut School of Medicine Farmington Connecticut USA

2. Department of Neurology Johns Hopkins University School of Medicine Baltimore Maryland USA

3. Department of Neuropediatrics, Developmental Neurology and Social Pediatrics, LMU Campus Innenstadt University of Munich Munich Germany

4. Department of Neurology UCLA Clinical and Translational Research Center Los Angeles California USA

5. Pediatric Neurology, Gillette Children's Specialty Healthcare St Paul Minnesota USA

6. Department of Neurology Seattle Children's Research Institute Seattle Washington USA

7. Department of Pediatrics, Neurology and Neurotherapeutics UT Southwestern Medical Center Dallas Texas USA

8. Biogen Cambridge Massachusetts USA

9. Biogen Maidenhead UK

Publisher

Wiley

Subject

Physiology (medical),Cellular and Molecular Neuroscience,Clinical Neurology,Physiology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/mus.27187

Reference28 articles.

1. The Neurobiology of Childhood Spinal Muscular Atrophy

2. Spinal Muscular Atrophy: A Clinical and Research Update

3. 209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7–9 November 2014, Heemskerk, The Netherlands

4. Spinal Muscular Atrophy: Clinical Classification and Disease Heterogeneity

5. Correlation between severity and SMN protein level in spinal muscular atrophy

Cited by 54 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders;Communications Medicine;2024-01-05

2. Is it ethical to reimburse “SMA Innovative Treatments” from government resources?;2023-12-29

3. Evaluation of Chemically Modified Nucleic Acid Analogues for Splice Switching Application;ACS Omega;2023-12-11

4. Gene-Based Therapy for the Treatment of Spinal Muscular Atrophy Types 1 and 2 : A Systematic Review and Meta-Analysis;2023-12-07

5. Study on the efficacy, safety, and biomarkers of nusinersen in type II and III spinal muscular atrophy in children;Frontiers in Pediatrics;2023-12-04