Zobrazeno 1 - 10
of 15
pro vyhledávání: '"Jena M Krueger"'
Autor:
Aravindhan Veerapandiyan, Anne M. Connolly, Katherine D. Mathews, Stanley Nelson, Craig McDonald, Richard S. Finkel, Vettaikorumakankav Vedanarayanan, Cuixia Tian, Susan Apkon, Julie A. Parsons, Jonathan H. Soslow, William Bryan Burnette, Kaitlin Y. Batley, Susan T. Iannaccone, Carolina Tesi Rocha, Kevin M. Flanigan, Diana Bharucha‐Goebel, Sarah Wright, Migvis Monduy, Simona Treidler, Ashutosh Kumar, Nancy L. Kuntz, Vamshi K. Rao, Rachel Schrader, Saunder M. Bernes, Vikki Ann Stefans, Jena M. Krueger, Marcia V. Felker, Omer Abdul Hamid, Arpita Lakhotia, Susan Matesanz, Partha S. Ghosh, Natalie Katz, Hoda Abdel‐Hamid, Chamindra G. Laverty, Bo Hoon Lee, Amy Harper, Leigh Ramos‐Platt, Diana Castro, Russell J. Butterfield, Crystal M. Proud, Craig M. Zaidman, Emma Ciafaloni
Publikováno v:
Annals of the Child Neurology Society, Vol 2, Iss 3, Pp 184-188 (2024)
Externí odkaz:
https://doaj.org/article/df1cf7b52fa3423cafb1b07b7a03d530
Autor:
Jena M Krueger, Douglas R Nordli, Jr
Publikováno v:
Pediatric Neurology Briefs, Vol 29, Iss 1 (2015)
Investigators from the Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago IL and the Yale School of Medicine, New Haven, CT have conducted a prospective cohort study of 613 children with newly diagnosed epilepsy to evaluate the occurre
Externí odkaz:
https://doaj.org/article/47c0575894b2432b903697e6630dea53
Autor:
Thomas O. Crawford, John W. Day, Darryl C. De Vivo, Jena M. Krueger, Eugenio Mercuri, Andres Nascimento, Amy Pasternak, Elena Stacy Mazzone, Tina Duong, Guochen Song, Jing L. Marantz, Scott Baver, Dongzi Yu, Lan Liu, Basil T. Darras
Publikováno v:
Frontiers in Neurology, Vol 15 (2024)
Background and purposeAt 12 months in the phase 2 TOPAZ study, treatment with apitegromab was associated with both an improved motor function in patients with Type 2 or 3 spinal muscular atrophy (SMA) and with a favorable safety profile. This manuscr
Externí odkaz:
https://doaj.org/article/463093cc41344076b301b431a80c1691
Autor:
Marian L. Henderson, Jacob K. Zieba, Xiaopeng Li, Daniel B. Campbell, Michael R. Williams, Daniel L. Vogt, Caleb P. Bupp, Yvonne M. Edgerly, Surender Rajasekaran, Nicholas L. Hartog, Jeremy W. Prokop, Jena M. Krueger
Publikováno v:
BioTech, Vol 13, Iss 1, p 1 (2024)
Gene therapy holds promise as a life-changing option for individuals with genetic variants that give rise to disease. FDA-approved gene therapies for Spinal Muscular Atrophy (SMA), cerebral adrenoleukodystrophy, β-Thalassemia, hemophilia A/B, retina
Externí odkaz:
https://doaj.org/article/60765cdb5a054fb2909e7b2b14f2b561
Autor:
Kevin A. Strauss, Michelle A. Farrar, Francesco Muntoni, Kayoko Saito, Jerry R. Mendell, Laurent Servais, Hugh J. McMillan, Richard S. Finkel, Kathryn J. Swoboda, Jennifer M. Kwon, Craig M. Zaidman, Claudia A. Chiriboga, Susan T. Iannaccone, Jena M. Krueger, Julie A. Parsons, Perry B. Shieh, Sarah Kavanagh, Sitra Tauscher-Wisniewski, Bryan E. McGill, Thomas A. Macek
Publikováno v:
Nature Medicine. 28:1381-1389
Abstract SPR1NT (NCT03505099) was a Phase III, multicenter, single-arm study to investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutations treated at ≤6 weeks of life. Here, we report f
Autor:
Kevin A. Strauss, Michelle A. Farrar, Francesco Muntoni, Kayoko Saito, Jerry R. Mendell, Laurent Servais, Hugh J. McMillan, Richard S. Finkel, Kathryn J. Swoboda, Jennifer M. Kwon, Craig M. Zaidman, Claudia A. Chiriboga, Susan T. Iannaccone, Jena M. Krueger, Julie A. Parsons, Perry B. Shieh, Sarah Kavanagh, Melissa Wigderson, Sitra Tauscher-Wisniewski, Bryan E. McGill, Thomas A. Macek
Publikováno v:
Nature Medicine. 28:1390-1397
Most children with biallelic SMN1 deletions and three SMN2 copies develop spinal muscular atrophy (SMA) type 2. SPR1NT (NCT03505099), a Phase III, multicenter, single-arm trial, investigated the efficacy and safety of onasemnogene abeparvovec for pre
Autor:
Jena M. Krueger, Douglas R. Nordli
Publikováno v:
Pediatric Neurology Briefs, Vol 29, Iss 9 (2015)
Investigators from The Barrow Neurological Institute, Creighton University, University of Kentucky and the University of Calgary Faculty of Medicine investigated the effect of ketone bodies and the ketogenic diet on epileptic Kcna1-null mice.
Externí odkaz:
https://doaj.org/article/320880cb7d964579b52871db017d42f7
Autor:
Jena M. Krueger
Publikováno v:
Pediatric Neurology Briefs
Our aim was to identify clinical outcomes, serological features and possible prognostic indicators of paediatric myasthenia gravis (MG). We collected 74 MG patients with disease onset before the age of 16 years (73% pre-pubertal onset defined as ≤1
Autor:
Francis G. Ogrinc, Michelle A. Farrar, Jennifer M. Kwon, James L’Italien, Kayoko Saito, Francesco Muntoni, Douglas M. Sproule, Imran Kausar, Susan T. Iannaccone, Claudia A. Chiriboga, Elaine Kernbauer, Meredith Schultz, Hugh J. McMillan, Kevin A. Strauss, Jena M. Krueger, Kathryn J. Swoboda, Bryan E. McGill, Mariacristina Scoto, Julie A. Parsons, Sarah Kavanagh, Marcia Farrow, Sidney A Spector, Douglas E. Feltner
Publikováno v:
Journal of Neurology, Neurosurgery & Psychiatry. 90:e7.1-e7
BackgroundOnasemnogene abeparvovec (AVXS-101) is a gene-replacement therapy that treats the genetic root cause of SMA, biallelic survival motor neuron 1 gene (SMN1) deletion/mutation. In a phase 1 study, AVXS-101 improved survival and motor function
Autor:
Cynthia V. Stack, Charu Venkatesan, John Millichap, Jena M. Krueger, David G. Ritacco, Douglas R. Nordli, Srishti Nangia
Publikováno v:
Journal of Child Neurology. 31:547-552
Intracranial hemorrhage accounts for about 50% of all pediatric stroke. Studies of term infants with intracranial hemorrhage have shown favorable motor and cognitive outcome. The goal of this study was to examine the risk of developing epilepsy in fu