Valeria Calbi

Milan, Italy

Last Updated: 09/26/2026

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Found 38 publications

Cross-Correction in HSC Gene Therapy for Metachromatic Leukodystrophy.

Cross-Correction in HSC Gene Therapy for Metachromatic Leukodystrophy.

Journal: The New England journal of medicine

Published: July 29, 2026

Busulfan exposure variability and acute toxicity in pediatric patients undergoing hematopoietic stem cell gene therapy.

Busulfan exposure variability and acute toxicity in pediatric patients undergoing hematopoietic stem cell gene therapy.

Journal: Frontiers in pharmacology

Published: May 04, 2026

Preliminary Data of the First Year of Newborn Screening for Metachromatic Leukodystrophy (MLD) in Lombardy.

Preliminary Data of the First Year of Newborn Screening for Metachromatic Leukodystrophy (MLD) in Lombardy.

Journal: International journal of neonatal screening

Published: April 30, 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

Metachromatic Leukodystrophy: New Therapy Advancements and Emerging Research Directions.

Metachromatic Leukodystrophy: New Therapy Advancements and Emerging Research Directions.

Journal: Neurology

Published: June 27, 2025

Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy.

Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy.

Journal: The New England journal of medicine

Published: April 23, 2025

Key lessons from the first international treatment eligibility committee: the case of metachromatic leukodystrophy.

Key lessons from the first international treatment eligibility committee: the case of metachromatic leukodystrophy.

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

Published: March 31, 2025

Newborn Screening for Metachromatic Leukodystrophy in Tuscany: The Paradigm of a Successful Preventive Medicine Program.

Newborn Screening for Metachromatic Leukodystrophy in Tuscany: The Paradigm of a Successful Preventive Medicine Program.

Journal: International journal of neonatal screening

Published: March 11, 2025

Effects of atidarsagene autotemcel gene therapy on peripheral nerves in late-infantile metachromatic leukodystrophy.

Effects of atidarsagene autotemcel gene therapy on peripheral nerves in late-infantile metachromatic leukodystrophy.

Journal: Brain : a journal of neurology

Published: January 20, 2025

Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.

Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.

Journal: Science translational medicine

Published: May 01, 2024

Newborn screening in metachromatic leukodystrophy - European consensus-based recommendations on clinical management.

Newborn screening in metachromatic leukodystrophy - European consensus-based recommendations on clinical management.

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

Published: January 26, 2024

Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries.

Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries.

Journal: Orphanet journal of rare diseases

Published: November 14, 2023

Showing 1-12 of 38

Last Updated: 09/26/2026