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

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

Saved in:

Bibliographic Details
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
Tags:	Add Tag No Tags, Be the first to tag this record!

Similar Items

Abstract 008: Morphomechanical Analysis of Saccular Aneurysms
by: Andres Gudino, et al.
Published: (2023-11-01)

OOxytetracycline Hydrochloride (OTC-HCl) Application for Control of Palm Phytoplasmas
by: Brian W. Bahder, et al.
Published: (2019-04-01)

OOxytetracycline Hydrochloride (OTC-HCl) Application for Control of Palm Phytoplasmas
by: Brian W. Bahder, et al.
Published: (2019-04-01)

The Rationale for FK 506 in Inflammatory Bowel Disease
by: James C Reynolds, et al.
Published: (1993-01-01)

Examining the evolution and impact of OTC vending machines in Global Healthcare Systems
by: Ammar Abdulrahman Jairoun, et al.
Published: (2024-12-01)

Ruricius of Limoges, Caesarius of Arles, and Council of Agde 506
by: Elena Litovchenko
Published: (2022-12-01)

Titanium vanadium nickel, TiV0.08Ni0.92
by: Huizi Liu, et al.
Published: (2025-02-01)

Perioperative care in pediatric OTC deficiency and goldenhar syndrome: a case report
by: Daniela Torres Salazar, et al.
Published: (2025-05-01)

OTC gene duplication as the possible cause of massive hyperammonaemia with a fatal prognosis
by: Borkowska Natalia, et al.
Published: (2024-12-01)

Molecular Evolution of the Vertebrate FK506 Binding Protein 25
by: Fei Liu, et al.
Published: (2014-01-01)

Síndrome intra-selar: análise de 506 cirurgias transesfenoidais
by: Pedro Sampaio
Published: (1985-12-01)

EFEKTIVITAS PELATIHAN DAN INTERNSHIP OTC BALI UNTUK KOMPETENSI TENAGA KERJA PARIWISATA
by: PEP. Redyanti, et al.
Published: (2025-01-01)

ST8-t008-SCCmec IV methicillin-resistant Staphylococcus aureus in retail fresh cheese
by: Carolina Chaves, et al.
Published: (2024-11-01)

Phytoremediation of OTC-Cu/Zn contaminated sediment: Synergetic removal mechanism and microbial community response
by: Xueqi Zhang, et al.
Published: (2025-07-01)

Research on the selection of OTC drug supply chain sales models: based on medical insurance policy
by: Heng Wei, et al.
Published: (2025-02-01)

Successful Treatment of Acanthamoeba Keratitis According to New Protocol with Polihexanide 0.08% Therapy: Case Report
by: Tomislav Kuzman, et al.
Published: (2025-04-01)

Polihexanide (PHMB) 0.08% versus currently used treatments for Acanthamoeba keratitis: indirect treatment comparisons
by: Vincenzo Papa, et al.
Published: (2025-07-01)

Association of dropout history and HbA1c levels with subsequent dropout risk in patients with diabetes: a secondary analysis of the Japan Diabetes Outcome Intervention Trial-2 Large-Scale Trial 008 (J-DOIT2-LT008)
by: Katsuya Yamazaki, et al.
Published: (2025-08-01)

Factors influencing muslims’ purchase intention of halal-certified over-the-counter (OTC) medicines in Bekasi, Indonesia
by: Fitry Oktavia Fatmi, et al.
Published: (2024-06-01)

Development of an In Vitro Method for Assessing the Potential Irritation of Medical Devices and OTC Products Used in the Oral Cavity
by: Christian Pellevoisin, et al.
Published: (2025-03-01)

A Prospective Six-Month Study of Chronic Pain Sufferers: A Novel OTC Neuromodulation Therapy
by: Richard Staelin, et al.
Published: (2019-01-01)

Analisis Faktor Risiko Kejadian Hipertensi Pada Masyarakat Di RW 008 Kampung Areman, Kota Depok
by: Putri Paqita Vidiningsih, et al.
Published: (2022-09-01)

Synergistic Antitumor Effect of Doxorubicin and Tacrolimus (FK506) on Hepatocellular Carcinoma Cell Lines
by: Francesca Capone, et al.
Published: (2014-01-01)

Awareness, Attitudes, and Practices Regarding OTC Drugs in Circassian and Chechen Communities in Jordan: A Cross-Sectional Study
by: Qnais E, et al.
Published: (2025-06-01)

The Relationship of Over the Counter (OTC) Facial Soap Usage and Acne Risk in Sebelas Maret University Medical Students
by: Marsyanda Jalasena Mysea, et al.
Published: (2024-11-01)

Resilience of Spanish Firms: a comparative analysis of large and small businesses in the face of 008 financial crisis and COVID-19
by: Jose Sevilla, et al.
Published: (2025-01-01)

P065: Canonical splice variant c.298+2T>C in OTC associated with attenuated ornithine transcarbamylase deficiency
by: Janhawi Kelkar, et al.
Published: (2025-01-01)

Urea Cycle Defects: Early-Onset Disease Associated with A208T Mutation in OTC Gene—Expanding the Clinical Phenotype
by: Ana Isabel Sánchez, et al.
Published: (2017-01-01)

Qualitative study exploring the key determinants of information gathering to inform the management of over-the-counter (OTC) consultations in community pharmacies
by: Rumana Newlands, et al.
Published: (2019-08-01)

Status and influencing factors of OTC medicine use for self-medication in cold and cough: a cross-sectional survey in Japan
by: Yu-Shi Tian, et al.
Published: (2025-05-01)

Interfacial Polarization Switching in Al0.92Sc0.08N/GaN Heterostructures Grown by Sputter Epitaxy
by: Niklas Wolff, et al.
Published: (2025-08-01)

FK506 and Cyclosporin A Enhance IL-6 Production in Monocytes: A single-Cell Assay
by: K. Murayama, et al.
Published: (1994-01-01)

Phage p1-derived artificial chromosomes facilitate heterologous expression of the FK506 gene cluster.
by: Adam C Jones, et al.
Published: (2013-01-01)

Regulatory orchestration of FK506 biosynthesis in Streptomyces tsukubaensis NRRL 18488 revealed through systematic analysis
by: Namil Lee, et al.
Published: (2025-06-01)

506 Evaluating prediction models for conversion of clinically isolated syndrome to multiple sclerosis: A systematic review
by: Mei-An Nolan, et al.
Published: (2025-04-01)

A Comparative Study of Safety and Efficacy of Mitomycin-C (0.08 mg) and Bevacizumab (7.5 mg) in Cases of External Dacryocystorhinostomy
by: Sumit Kumar Sahu
Published: (2023-04-01)

Why patients fail to seek information on OTC product interactions with a direct-acting oral anticoagulant: perspectives on information-seeking
by: Marley P.D. Magee, et al.
Published: (2025-02-01)

Perceptions amongst medication users regarding South African patient package inserts (PPIs) of scheduled and over-the-counter (OTC) medications
by: Daleen Krige, et al.
Published: (2022-10-01)

Tacrolimus (FK506) promotes placentation and maternal-fetal tolerance through modulating FASN-CEACAM1 pathway
by: Xinhang Meng, et al.
Published: (2025-02-01)

The Immunosuppressant Tacrolimus (FK506) Facing the 21st Century: Past Findings, Present Applications and Future Trends
by: Carlos Barreiro, et al.
Published: (2024-12-01)