Combination molecular therapies for type 1 spinal muscular atrophy
2020 · Muscle & Nerve · 78 citations
https://doi.org/10.1002/mus.27034
V
Vamshi K. Rao
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Vamshi K. Rao is an academic researcher from Northwestern University. The author has contributed to research in topics: Muscle Physiology and Disorders & Neurogenetic and Muscular Disorders Research & Congenital Anomalies and Fetal Surgery. The author has an h-index of 15, co-authored 53 publications.
ORCID: 0000-0002-0130-2292Papers by this author
worksAxonal polyneuropathy and ataxia in children: consider Perrault Syndrome, a case report
2023 · BMC Medical Genomics · 5 citations
https://doi.org/10.1186/s12920-023-01599-4
A case report of riboflavin transporter deficiency: A novel heterozygous pathogenic variant in the SLC52A3 gene
2024 · Molecular Genetics and Metabolism Reports · 2 citations
https://doi.org/10.1016/j.ymgmr.2024.101051
New Clinical Phenotype in a Child Presenting With an <i>FHL1</i> Mutation
2025 · Journal of Child Neurology · 0 citations
https://doi.org/10.1177/08830738251332749
Differences in swallowing efficacy of disease modifying treatment between infants receiving pre-symptomatic and symptomatic administration
2025 · Orphanet Journal of Rare Diseases · 0 citations
https://doi.org/10.1186/s13023-025-04049-9
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