Nicholas J.C. Smith - Pediat Neurol, Womens & Childrens Hosp

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论文共 24 篇作者统计合作学者相似作者

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Evaluation of Mobilization, Apheresis, and Conditioning Regimen and Engraftment in Patients Receiving One-Time Gene Therapy with Elivaldogene Autotemcel (Eli-Cel) for Cerebral Adrenoleukodystrophy (CALD)

Christine N. Duncan,Paul J. Orchard,Florian S. Eichler,Jörn-Sven Kühl,Satiro N. De Oliveira,Adrian J. Thrasher, Robert Chiesa,Jean-Hugues Dalle,Ami J. Shah,Franco Locatelli,Nicholas J.C. Smith,Juliana Folloni Fernandes,Hernán M. Amartino,Caroline A. Lindemans, Jack Zhao, Ajay Singh,Himal L. Thakar,Vinod K. Prasad,Andrew C. Dietz,David A. Williams

Transplantation and Cellular Therapyno. 2 (2024): S232-S233

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The collective burden of childhood dementia: a scoping review

Kristina L. Elvidge,John Christodoulou,Michelle A. Farrar,Dominic Tilden,Megan Maack,Madeline Valeri,Magda Ellis,Nicholas J. C. Smith

BRAINno. 11 (2023): 4446-4455

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Interim Results of Transpher A, a Multicenter, Single-Dose Clinical Trial of UX111 Gene Therapy for Sanfilippo Syndrome Type A (mucopolysaccharidosis IIIA)

Kevin M. Flanigan,Nicholas Smith,Maria Luz Couce,Deepa Rajan,Kristen Truxal,Kim L. McBride,Maria Jose de Castro Lopez,Maria Fuller,John Taylor,Ana B. Del Campo, Igor Grachev,Heather Lau

Molecular genetics and metabolismno. 2 (2023): 107101-107101

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Variants in the imprinted gene MAGEL2

Evelyn Douglas,Louisa Sanchez,Jessica Burdett, Shannon Le Blanc,Nicholas J. C. Smith,Andrew Dubowsky,David Lawrence,Karin Kassahn,Sui Yu,Kathie Friend

EUROPEAN JOURNAL OF HUMAN GENETICS（2022）

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Sanfilippo Syndrome: Consensus Guidelines for Clinical Care

Nicole Muschol,Roberto Giugliani,Simon A. Jones,Joseph Muenzer,Nicholas J. C. Smith,Chester B. Whitley, Megan Donnell,Elise Drake,Kristina Elvidge,Lisa Melton,Cara O’Neill, MPS III Guideline Development Group

Orphanet Journal of Rare Diseases（2022）

引用6浏览0引用

Oligonucleotide correction of an intronic TIMMDC1 variant in cells of patients with severe neurodegenerative disorder

Raman Kumar,Mark A. Corbett,Nicholas J. C. Smith,Daniella H. Hock,Zoya Kikhtyak,Liana N. Semcesen,Atsushi Morimoto,Sangmoon Lee,David A. Stroud,Joseph G. Gleeson,Eric A. Haan,Jozef Gecz

NPJ GENOMIC MEDICINE（2022）

引用8浏览0WOSNATURE引用

Severely Impaired CTL Killing is a Feature of the Neurological Disorder Niemann-Pick Disease Type C1

Daniela Castiblanco,Jesse A. Rudd-Schmidt,Tahereh Noori,Vivien R. Sutton,Ya Hui Hung,Thijs W. H. Flinsenberg,Adrian W. Hodel,Neil D. Young,Nicholas Smith,Drago Bratkovic,Heidi Peters,Mark Walterfang,Joseph A. Trapani,Amelia J. Brennan,Ilia Voskoboinik

Blood（2022）

引用6浏览0WOS引用

Disease Stabilization Following Treatment with elivaldogene autotemcel (eli-cel, Lenti-D) Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy: Interim Results from Phase 2/3 (ALD-102) and Phase 3 (ALD-104) Studies (2064)

Florian Eichler,Christine Duncan,Paul J. Orchard,Satiro N. De Oliveira,Adrian J. Thrasher,Caroline Sevin,Hernan Amartino,Nicholas J.C. Smith,Jörn-Sven Kühl,Patricia L. Musolino,Daniel L. Kenney-Jung,Robert Chiesa,Jean-Hugues Dalle, Shuang He,Andrew C. Dietz,Elizabeth McNeil,David R. Williams

Neurology（2021）

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