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Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment

Spinal muscular atrophy (SMA) is the most common genetic disease affecting infants and young adults. Due to mutation/deletion of the survival motor neuron (SMN) gene, SMA is characterized by the SMN protein lack, resulting in motor neuron impairment, skeletal muscle atrophy and premature death. Even...

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Detalhes bibliográficos
Publicado no:Front Pharmacol
Main Authors: Menduti, Giovanna, Rasà, Daniela Maria, Stanga, Serena, Boido, Marina
Formato: Artigo
Idioma:Inglês
Publicado em: Frontiers Media S.A. 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7689316/
https://ncbi.nlm.nih.gov/pubmed/33281605
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fphar.2020.592234
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