Decision-making and challenges within the evolving treatment algorithm in spinal muscular atrophy: a clinical perspective-Reference-Cited by-同舟云学术

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Decision-making and challenges within the evolving treatment algorithm in spinal muscular atrophy: a clinical perspective

Published:2023-06 Issue:7 Volume:23 Page:571-586
ISSN:1473-7175
Container-title:Expert Review of Neurotherapeutics
language:en
Short-container-title:Expert Review of Neurotherapeutics

Author:

Balaji Lakshmi¹²,Farrar Michelle A¹²³,D’Silva Arlene M¹²³,Kariyawasam Didu S¹²^ORCID

Affiliation:

1. Department of Neurology, Sydney Children’s Hospital Network, Sydney, New South Wales, Australia

2. Discipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health UNSW, Sydney, Australia

3. UNSW Kensington Campus, Sydney, Australia

Funder

National Health and Medical Research Council

University of New South Wales

Publisher

Informa UK Limited

Subject

Pharmacology (medical),Neurology (clinical),General Neuroscience

Link

https://www.tandfonline.com/doi/pdf/10.1080/14737175.2023.2218549

Reference126 articles.

1. Identification and characterization of a spinal muscular atrophy-determining gene

2. Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?;Burghes AH;Nat Rev Neurosci,2009

3. A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2

4. 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;Feldkötter M;Am J Hum Genet,2002

5. Spinal muscular atrophy: in the challenge lies a solution;Wirth B;Trends Neurosci,2021

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1. Gene therapy-based strategies for spinal muscular atrophy—an Asia-Pacific perspective;Molecular and Cellular Pediatrics;2023-11-15

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