With the advent of disease-modifying therapies for spinal muscular atrophy, prenatal and extra-neural alterations associated with the condition have garnered increasing attention as potential determinants of the therapeutic window and efficacy of novel drugs. Two recent studies highlight the impact of spinal muscular atrophy on prenatal bone and organ development.
Spinal muscular atrophy — challenges in the therapeutic era / I. Faravelli, S. Corti. - In: NATURE REVIEWS. NEUROLOGY. - ISSN 1759-4758. - (2020). [Epub ahead of print] [10.1038/s41582-020-00411-6]
Spinal muscular atrophy — challenges in the therapeutic era
I. Faravelli;S. Corti
2020
Abstract
With the advent of disease-modifying therapies for spinal muscular atrophy, prenatal and extra-neural alterations associated with the condition have garnered increasing attention as potential determinants of the therapeutic window and efficacy of novel drugs. Two recent studies highlight the impact of spinal muscular atrophy on prenatal bone and organ development.File in questo prodotto:
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