The Identification of Novel Biomarkers Is Required to Improve Adult SMA Patient Stratification, Diagnosis and Treatment

The Identification of Novel Biomarkers Is Required to Improve Adult SMA Patient Stratification, Diagnosis and Treatment

Spinal muscular atrophy (SMA) is currently classified into five different subtypes, from the most severe (type 0) to the mildest (type 4) depending on age at onset, best motor function achieved, and copy number of the <i>SMN2</i> gene. The two recent approved treatments for SMA patients...

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Bibliographic Details
Main Authors: Piera Smeriglio, Paul Langard, Giorgia Querin, Maria Grazia Biferi
Format: Article
Language:English
Published: MDPI AG 2020-07-01
Series:Journal of Personalized Medicine
Subjects:
spinal muscular atrophy
adult patients
disease heterogeneity
Nusinersen
disease modifiers
functional outcomes
Online Access:https://www.mdpi.com/2075-4426/10/3/75

Internet

https://www.mdpi.com/2075-4426/10/3/75

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