Spinal Muscular Atrophy: An Evolving Scenario through New Perspectives in Diagnosis and Advances in Therapies

Spinal Muscular Atrophy: An Evolving Scenario through New Perspectives in Diagnosis and Advances in Therapies

Spinal muscular atrophy (SMA) linked to 5q is a recessive motor neuron disease characterized by progressive and diffuse weakness and muscular atrophy. SMA is the most common neurodegenerative disease in childhood with an incidence of approximately 1 in 6000–10,000 live births, being long considered...

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Bibliographic Details
Main Authors: Ilaria Angilletta, Rossella Ferrante, Roberta Giansante, Lucia Lombardi, Alessandra Babore, Anastasia Dell’Elice, Elisa Alessandrelli, Stefania Notarangelo, Marianna Ranaudo, Claudia Palmarini, Vincenzo De Laurenzi, Liborio Stuppia, Claudia Rossi
Format: Article
Language:English
Published: MDPI AG 2023-10-01
Series:International Journal of Molecular Sciences
Subjects:
spinal muscular atrophy
gene therapy
newborn screening
disease-modifying therapies
diagnosis
psychological adjustment
Online Access:https://www.mdpi.com/1422-0067/24/19/14873

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