Day, John W.

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

Mercuri, E., Darras, B. T., Chiriboga, C. A., Day, J. W., Campbell, C., Connolly, A. M., Iannaccone, S. T., Kirschner, J., Kuntz, N. L., Saito, K., et al. (2018) · New England Journal of Medicine

Neurogenetic and Muscular Disorders ResearchLysosomal Storage Disorders Research

Cited by 1,433

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

Day, J. W., Finkel, R. S., Chiriboga, C. A., Connolly, A. M., Crawford, T. O., Darras, B. T., Iannaccone, S. T., Kuntz, N. L., Peña, L. D. M., Shieh, P. B., et al. (2021) · The Lancet Neurology

Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy research

Cited by 494

MBNL Sequestration by Toxic RNAs and RNA Misprocessing in the Myotonic Dystrophy Brain

Goodwin, M., Mohan, A., Batra, R., Lee, K.-Y., Charizanis, K., Gómez, F. J. F., Eddarkaoui, S., Sergeant, N., Buée, L., Kimura, T., et al. (2015) · Cell Reports

RNA Research and SplicingGenetic Neurodegenerative Diseases

Cited by 151

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

Day, J. W., Mendell, J. R., Mercuri, E., Finkel, R. S., Strauss, K. A., Kleyn, A., Tauscher-Wisniewski, S., Tukov, F. F., Reyna, S. P., & Chand, D. H. (2021) · Drug Safety

Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy research

Cited by 119

Telomere shortening is a hallmark of genetic cardiomyopathies

Chang, A. C. Y., Chang, A. C. H., Kirillova, A., Sasagawa, K., Su, W., Weber, G., Lin, J., Termglinchan, V., Karakikes, I., Seeger, T., et al. (2018) · Proceedings of the National Academy of Sciences

Telomeres, Telomerase, and SenescenceCardiomyopathy and Myosin Studies

Cited by 91

High frequency of gastrointestinal manifestations in myotonic dystrophy type 1 and type 2

Hilbert, J. E., Barohn, R. J., Clemens, P. R., Luebbe, E. A., Martens, W. B., McDermott, M. P., Parkhill, A. L., Tawil, R., Thornton, C. A., Moxley, R. T., et al. (2017) · Neurology

Genetic Neurodegenerative DiseasesGastrointestinal motility and disorders

Cited by 70

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

Cited by 48