Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy-Reference-Cited by-同舟云学术

Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy

Published:2021-07-20 Issue:4 Volume:64 Page:413-427
ISSN:0148-639X
Container-title:Muscle & Nerve
language:en
Short-container-title:Muscle & Nerve

Author:

Kichula Elizabeth A.¹,Proud Crystal M.²,Farrar Michelle A.³,Kwon Jennifer M.⁴,Saito Kayoko⁵,Desguerre Isabelle⁶,McMillan Hugh J.⁷

Affiliation:

1. Children's Hospital of Philadelphia Philadelphia Pennsylvania USA

2. Children's Hospital of the King's Daughters Norfolk Virginia USA

3. School of Women's and Children's Health, UNSW Medicine University of New South Wales Sydney and Sydney Children's Hospital Network Sydney New South Wales Australia

4. School of Medicine and Public Health University of Wisconsin Madison Wisconsin USA

5. Institute of Medical Genetics, Tokyo Women's Medical University Tokyo Japan

6. Necker‐Enfants Malades Hospital University of Paris, AP‐HP Paris France

7. Children's Hospital of Eastern Ontario University of Ottawa Ottawa Ontario Canada

Publisher

Wiley

Subject

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

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/mus.27363

Reference87 articles.

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2. Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72 400 specimens

3. Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2

4. Correlation between SMA type and SMN2 copy number revisited: An analysis of 625 unrelated Spanish patients and a compilation of 2834 reported cases

5. Quantitative Analyses of SMN1 and SMN2 Based on Real-Time LightCycler PCR: Fast and Highly Reliable Carrier Testing and Prediction of Severity of Spinal Muscular Atrophy

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