An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy-Reference-Cited by-同舟云学术

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An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy

Published:2023-03-01 Issue:5-6 Volume:34 Page:180-191
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Li Yueyi¹,Zeng Hongyu¹²,Wei Yuhao¹²,Ma Xuelei¹,He Zhiyao¹³^ORCID

Affiliation:

1. Division of Biotherapy, Cancer Center, West China Hospital, Sichuan University, Chengdu, China.

2. West China School of Medicine, West China Hospital, Sichuan University, Chengdu, China.

3. Department of Pharmacy, West China Hospital, Sichuan University, Chengdu, China.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2022.189

Reference102 articles.

1. Technical standards and guidelines for spinal muscular atrophy testing

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

3. 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

4. Nusinersen: First Global Approval

5. Onasemnogene Abeparvovec: First Global Approval

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1. Health information literacy among children with spinal muscular atrophy and their caregivers;Italian Journal of Pediatrics;2024-08-26

2. Clinical perspectives: Treating spinal muscular atrophy;Molecular Therapy;2024-08

3. The yield of genetic workup for middle-aged and elderly patients with neurological disorders in a real-world setting;Journal of the Neurological Sciences;2024-08

4. Quantitative proteomics unveils known and previously unrecognized alterations in neuropathic nerves;Journal of Neurochemistry;2024-07-29

5. Gene therapy offers promise, but timing is crucial for SMA treatment;European Journal of Paediatric Neurology;2024-07

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