New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges

Spinal muscular atrophy (SMA) is one of the most common autosomal recessive diseases with progressive weakness of skeletal and respiratory muscles, leading to significant disability. The disorder is caused by mutations in the survival motor neuron 1 (<i>SMN1</i>) gene and a consequent de...

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Bibliographic Details
Main Authors: Sonia Messina, Maria Sframeli
Format: Article
Language:English
Published: MDPI AG 2020-07-01
Series:Journal of Clinical Medicine
Subjects:
Online Access:https://www.mdpi.com/2077-0383/9/7/2222

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