McColly, Markus

Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy

Mendell, J. R., Al-Zaidy, S. A., Lehman, K. J., McColly, M., Lowes, L. P., Alfano, L. N., Reash, N. F., Iammarino, M. A., Church, K. R., Kleyn, A., et al. (2021) · JAMA Neurology

Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy 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

Cited by 179