Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy
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- 13 March 2025
- journal article
- Published by Massachusetts Medical Society in New England Journal of Medicine
- Vol. 392 (11) , 1138-1140
Abstract
No abstract availableThis publication has 4 references indexed in Scilit:
- Spinal muscular atrophyNature Reviews Disease Primers, 2022
- Implications of circulating neurofilaments for spinal muscular atrophy treatment early in life: A case seriesMolecular Therapy - Methods & Clinical Development, 2021
- Risdiplam in Type 1 Spinal Muscular AtrophyNew England Journal of Medicine, 2021
- Correlation between SMA type and SMN2 copy number revisited: An analysis of 625 unrelated Spanish patients and a compilation of 2834 reported casesNeuromuscular Disorders, 2018