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: | , , , , , , , , , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2025-01-01
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| Series: | Genetics in Medicine Open |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S294977442500891X |
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| _version_ | 1850178258661801984 |
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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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