Data di Pubblicazione:
2020
Citazione:
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]
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.
Tipologia IRIS:
01 - Articolo su periodico
Keywords:
nusinersen
Elenco autori:
I. Faravelli, S. Corti
Link alla scheda completa: