Shieh, Perry B.

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

Identification of a Signaling Pathway Involved in Calcium Regulation of BDNF Expression

Shieh, P. B., Hu, S.-C., Bobb, K., Timmusk, T., & Ghosh, A. (1998) · Neuron

Nerve injury and regenerationNeuroscience and Neuropharmacology Research

Cited by 693

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

Mutations in the RNA exosome component gene EXOSC3 cause pontocerebellar hypoplasia and spinal motor neuron degeneration

Wan, J., Yourshaw, M., Mamsa, H., Rudnik-Schöneborn, S., Menezes, M. P., Hong, J. E., Leong, D. W., Senderek, J., Salman, M. S., Chitayat, D., et al. (2012) · Nature Genetics

Fetal and Pediatric Neurological DisordersGenetics and Neurodevelopmental Disorders

Cited by 257

Cerebellar transcranial magnetic stimulation impairs verbal working memory

Desmond, J. E., Chen, S. H. A., & Shieh, P. B. (2005) · Annals of Neurology

Transcranial Magnetic Stimulation StudiesNeural and Behavioral Psychology Studies

Cited by 141

Re:”Moving forward after two deaths in a gene therapy trial of myotubular myopathy” by Wilson and flotte

Shieh, P. B., Bönnemann, C. G., Müller-Felber, W., Blaschek, A., Dowling, J. J., Kuntz, N. L., & Seferian, A. M. (2020) · Human Gene Therapy

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 91