P008: A first in human, single arm, open label phase 1/2 study evaluating ECUR-506 in neonatal OTC Deficiency: Initial Observations

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Main Authors: Julien Baruteau, Gabriel Cohn, Anil Dhawan, Anupam Chakrapani, Stephanie Grunewald, Molly Abbott, Helen Ashton, Sophie Foxall, Ai Ling Koh, Christos Lazaridis, Havea Navarro-Kennedy, Hamza Patel, Siyamini Sivananthan, Eleni Tamvaki, Katy Vecchiato, Matthew Hall, Karen Kuhn, Thomas White, Barbara Pinho, George Diaz
Format: Article
Language:English
Published: Elsevier 2025-01-01
Series:Genetics in Medicine Open
Online Access:http://www.sciencedirect.com/science/article/pii/S294977442500891X
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author Julien Baruteau
Gabriel Cohn
Anil Dhawan
Anupam Chakrapani
Stephanie Grunewald
Molly Abbott
Helen Ashton
Sophie Foxall
Ai Ling Koh
Christos Lazaridis
Havea Navarro-Kennedy
Hamza Patel
Siyamini Sivananthan
Eleni Tamvaki
Katy Vecchiato
Matthew Hall
Karen Kuhn
Thomas White
Barbara Pinho
George Diaz
author_facet Julien Baruteau
Gabriel Cohn
Anil Dhawan
Anupam Chakrapani
Stephanie Grunewald
Molly Abbott
Helen Ashton
Sophie Foxall
Ai Ling Koh
Christos Lazaridis
Havea Navarro-Kennedy
Hamza Patel
Siyamini Sivananthan
Eleni Tamvaki
Katy Vecchiato
Matthew Hall
Karen Kuhn
Thomas White
Barbara Pinho
George Diaz
author_sort Julien Baruteau
collection DOAJ
format Article
id doaj-art-5b51ed83688d4b17ab021d5ebd07f52a
institution OA Journals
issn 2949-7744
language English
publishDate 2025-01-01
publisher Elsevier
record_format Article
series Genetics in Medicine Open
spelling doaj-art-5b51ed83688d4b17ab021d5ebd07f52a2025-08-20T02:18:47ZengElsevierGenetics in Medicine Open2949-77442025-01-01310285210.1016/j.gimo.2025.102852P008: A first in human, single arm, open label phase 1/2 study evaluating ECUR-506 in neonatal OTC Deficiency: Initial ObservationsJulien Baruteau0Gabriel Cohn1Anil Dhawan2Anupam Chakrapani3Stephanie Grunewald4Molly Abbott5Helen Ashton6Sophie Foxall7Ai Ling Koh8Christos Lazaridis9Havea Navarro-Kennedy10Hamza Patel11Siyamini Sivananthan12Eleni Tamvaki13Katy Vecchiato14Matthew Hall15Karen Kuhn16Thomas White17Barbara Pinho18George Diaz19Great Ormond Street Hospital for Children, London, UK, UCL Great Ormond Street Institute of Child Health, London, UKiECURE, Inc, Blue Bell, PAKing's College Hospital, London, UKGreat Ormond Street Hospital for Children, London, UKGreat Ormond Street Hospital for Children, London, UKGreat Ormond Street Hospital for Children, London, UKGreat Ormond Street Hospital for Children, London, UKGreat Ormond Street Hospital for Children, London, UKGreat Ormond Street Hospital for Children, London, UKGreat Ormond Street Hospital for Children, London, UKGreat Ormond Street Hospital for Children, London, UKGreat Ormond Street Hospital for Children, London, UKGreat Ormond Street Hospital for Children, London, UKGreat Ormond Street Hospital for Children, London, UKGreat Ormond Street Hospital for Children, London, UKiECURE, Inc, Blue Bell, PAiECURE, Inc, Blue Bell, PAiECURE, Inc, Blue Bell, PAFortrea, Inc, Princeton, NJiECURE, Inc, Blue Bell, PAhttp://www.sciencedirect.com/science/article/pii/S294977442500891X
spellingShingle Julien Baruteau
Gabriel Cohn
Anil Dhawan
Anupam Chakrapani
Stephanie Grunewald
Molly Abbott
Helen Ashton
Sophie Foxall
Ai Ling Koh
Christos Lazaridis
Havea Navarro-Kennedy
Hamza Patel
Siyamini Sivananthan
Eleni Tamvaki
Katy Vecchiato
Matthew Hall
Karen Kuhn
Thomas White
Barbara Pinho
George Diaz
P008: A first in human, single arm, open label phase 1/2 study evaluating ECUR-506 in neonatal OTC Deficiency: Initial Observations
Genetics in Medicine Open
title P008: A first in human, single arm, open label phase 1/2 study evaluating ECUR-506 in neonatal OTC Deficiency: Initial Observations
title_full P008: A first in human, single arm, open label phase 1/2 study evaluating ECUR-506 in neonatal OTC Deficiency: Initial Observations
title_fullStr P008: A first in human, single arm, open label phase 1/2 study evaluating ECUR-506 in neonatal OTC Deficiency: Initial Observations
title_full_unstemmed P008: A first in human, single arm, open label phase 1/2 study evaluating ECUR-506 in neonatal OTC Deficiency: Initial Observations
title_short P008: A first in human, single arm, open label phase 1/2 study evaluating ECUR-506 in neonatal OTC Deficiency: Initial Observations
title_sort p008 a first in human single arm open label phase 1 2 study evaluating ecur 506 in neonatal otc deficiency initial observations
url http://www.sciencedirect.com/science/article/pii/S294977442500891X
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