Menier, Melissa

AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort

Al-Zaidy, S. A., Kolb, S. J., Lowes, L., Alfano, L. N., Shell, R., Church, K. R., Nagendran, S., Sproule, D. M., Feltner, D. E., Wells, C., et al. (2019) · Journal of Neuromuscular Diseases

Neurogenetic and Muscular Disorders ResearchLysosomal Storage Disorders Research

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Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy

Lowes, L. P., Alfano, L. N., Arnold, W. D., Shell, R., Prior, T. W., McColly, M., Lehman, K. J., Church, K., Sproule, D. M., Nagendran, S., et al. (2019) · Pediatric Neurology

Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy research

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Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

Day, J. W., Finkel, R. S., Mercuri, E., Swoboda, K. J., Menier, M., van Olden, R., Tauscher-Wisniewski, S., & Mendell, J. R. (2021) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

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