Newborn screening facilitates early theranostics and improved spinal muscular atrophy outcome: five-year real-world evidence from Taiwan

Newborn screening facilitates early theranostics and improved spinal muscular atrophy outcome: five-year real-world evidence from Taiwan

Abstract Background Recent findings indicate that infants with spinal muscular atrophy (SMA) treated early through newborn screening (NBS) have better outcomes. This study aimed to investigate the long-term outcomes of a 5-year SMA NBS program in Taiwan. Results From September 2017 to August 2022, t...

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Main Authors: Chen-Hua Wang, Ting-Rong Hsu, Mei-Ying Liu, Li-Yun Wang, I-Jun Chou, Wang-Tso Lee, Wen-Chen Liang, Inn-Chi Lee, Hsiao-Jan Chen, Shu-Min Kao, Hui-Chen Ho, Dau-Ming Niu, Kwang-Jen Hsiao, Ming-Yuh Chang, Hui-Min Hsieh, Yuh-Jyh Jong
Format: Article
Language:English
Published: BMC 2025-04-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Spinal muscular atrophy
Newborn screening
Early treatment
Symptomatic treatment
Presymptomatic treatment
Disease-modifying therapy
Online Access:https://doi.org/10.1186/s13023-025-03697-1
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https://doi.org/10.1186/s13023-025-03697-1

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