Molecular Biomarkers for the Diagnosis, Prognosis, and Pharmacodynamics of Spinal Muscular Atrophy

Molecular Biomarkers for the Diagnosis, Prognosis, and Pharmacodynamics of Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a progressive degenerative illness that affects 1 in every 6 to 11,000 live births. This autosomal recessive disorder is caused by homozygous deletion or mutation of the <i>SMN1</i> gene (survival motor neuron). As a backup, the <i>SMN1</i> ge...

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Bibliographic Details
Main Authors: Marija Babić, Maria Banović, Ivana Berečić, Tea Banić, Mirjana Babić Leko, Monika Ulamec, Alisa Junaković, Janja Kopić, Jadranka Sertić, Nina Barišić, Goran Šimić
Format: Article
Language:English
Published: MDPI AG 2023-08-01
Series:Journal of Clinical Medicine
Subjects:
spinal muscular atrophy
survival motor neuron 1 protein
pharmacological biomarkers
prognosis
nusinersen
Online Access:https://www.mdpi.com/2077-0383/12/15/5060

Internet

https://www.mdpi.com/2077-0383/12/15/5060

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