Connolly, Anne M.

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Finkel, R. S., Mercuri, E., Darras, B. T., Connolly, A. M., Kuntz, N. L., Kirschner, J., Chiriboga, C. A., Saito, K., Servais, L., Tizzano, E., et al. (2017) · New England Journal of Medicine

Neurogenetic and Muscular Disorders ResearchAmyotrophic Lateral Sclerosis Research

Cited by 2,337

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

Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations

Mendell, J. R., Connolly, A. M., Lehman, K. J., Griffin, D. A., Khan, S. Z., Dharia, S. D., Quintana-Gallardo, L., & Rodino-Klapac, L. R. (2022) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 124

CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy

Escolar, D. M., Buyse, G., Henricson, E., Leshner, R., Florence, J., Mayhew, J., Tesi‐Rocha, C., Gorni, K., Pasquali, L., Patel, K. M., et al. (2005) · Annals of Neurology

Muscle Physiology and DisordersMuscle metabolism and nutrition

Cited by 107

Rapamycin nanoparticles target defective autophagy in muscular dystrophy to enhance both strength and cardiac function

Bibee, K. P., Cheng, Y., Ching, J. K., Marsh, J. N., Li, A. J., Keeling, R. M., Connolly, A. M., Golumbek, P. T., Myerson, J. W., Hu, G., et al. (2014) · The FASEB Journal

Muscle Physiology and DisordersAutophagy in Disease and Therapy

Cited by 83