Arunabha Ghosh

Oxford Road
Manchester, ENG, United Kingdom

Last Updated: 09/26/2026

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Found 30 publications

The future of hematopoietic stem cell and stem cell gene therapy for metabolic diseases.

The future of hematopoietic stem cell and stem cell gene therapy for metabolic diseases.

Journal: Regenerative medicine

Published: June 10, 2026

Efficacy and safety of pegzilarginase in patients below 2 years of age with arginase 1 deficiency: a phase 3, open-label, multi-centre study.

Efficacy and safety of pegzilarginase in patients below 2 years of age with arginase 1 deficiency: a phase 3, open-label, multi-centre study.

Journal: EClinicalMedicine

Published: March 27, 2026

European expert recommendations for comprehensive pre-treatment, treatment-phase and post-treatment care of patients with metachromatic leukodystrophy treated with autologous haematopoietic stem and progenitor cell gene therapy.

European expert recommendations for comprehensive pre-treatment, treatment-phase and post-treatment care of patients with metachromatic leukodystrophy treated with autologous haematopoietic stem and progenitor cell gene therapy.

Journal: European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society

Published: March 25, 2026

Clinical Characteristics of Arginase 1 Deficiency: Natural History Insights From International Clinical Trials.

Clinical Characteristics of Arginase 1 Deficiency: Natural History Insights From International Clinical Trials.

Journal: Journal of inherited metabolic disease

Published: September 03, 2025

Failure of Allogeneic Transplant to Correct Sialidosis Despite Early Diagnosis and Full Donor Engraftment of Non-Carrier Leucocytes.

Failure of Allogeneic Transplant to Correct Sialidosis Despite Early Diagnosis and Full Donor Engraftment of Non-Carrier Leucocytes.

Journal: Journal of inherited metabolic disease

Published: May 22, 2025

Does Early Diagnosis and Treatment Alter the Clinical Course of Wolman Disease? Divergent Trajectories in Two Siblings and a Consideration for Newborn Screening.

Does Early Diagnosis and Treatment Alter the Clinical Course of Wolman Disease? Divergent Trajectories in Two Siblings and a Consideration for Newborn Screening.

Journal: International journal of neonatal screening

Published: November 03, 2024

Retinopathy in Mucopolysaccharidoses.

Retinopathy in Mucopolysaccharidoses.

Journal: Ophthalmology

Published: August 08, 2024

Retrospective Review of Positive Newborn Screening Results for Isovaleric Acidemia and Development of a Strategy to Improve the Efficacy of Newborn Screening in the UK.

Retrospective Review of Positive Newborn Screening Results for Isovaleric Acidemia and Development of a Strategy to Improve the Efficacy of Newborn Screening in the UK.

Journal: International journal of neonatal screening

Published: January 23, 2024

Twice weekly dosing with Sebelipase alfa (Kanuma®) rescues severely ill infants with Wolman disease.

Twice weekly dosing with Sebelipase alfa (Kanuma®) rescues severely ill infants with Wolman disease.

Journal: Orphanet journal of rare diseases

Published: April 18, 2023

Clinical, biochemical and molecular analysis in a cohort of individuals with gyrate atrophy.

Clinical, biochemical and molecular analysis in a cohort of individuals with gyrate atrophy.

Journal: Orphanet journal of rare diseases

Published: January 31, 2023

Prolidase deficiency in an infant with an incidental finding of methaemoglobinaemia.

Prolidase deficiency in an infant with an incidental finding of methaemoglobinaemia.

Journal: BMJ case reports

Published: November 19, 2021

Correction to: Long-term survival with sebelipase alfa enzyme replacement therapy in infants with rapidly progressive lysosomal acid lipase deficiency: final results from 2 open-label studies.

Correction to: Long-term survival with sebelipase alfa enzyme replacement therapy in infants with rapidly progressive lysosomal acid lipase deficiency: final results from 2 open-label studies.

Journal: Orphanet journal of rare diseases

Published: March 02, 2021

Showing 1-12 of 30

Last Updated: 09/26/2026