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Last Updated: 10/31/2025
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Found 1584 publications
Evidence Regarding Metachromatic Leukodystrophy Newborn Screening.
Journal: Pediatrics
Published: August 08, 2025
Metachromatic Leukodystrophy: New Therapy Advancements and Emerging Research Directions.
Journal: Neurology
Published: June 27, 2025
Exploration Into Lived Experiences of Multiple Sulfatase Deficiency-Affected Individuals and Their Families.
Journal: Journal of child neurology
Published: May 14, 2025
Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy.
Journal: The New England journal of medicine
Published: April 23, 2025
Encapsulated cells as an enzyme replacement therapy for metachromatic leukodystrophy.
Journal: Journal of controlled release : official journal of the Controlled Release Society
Published: April 14, 2025
Lenmeldy (atidarsagene autotemcel) for individuals with early metachromatic leukodystrophy (MLD): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).
Journal: Genetics in medicine open
Published: April 02, 2025
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: Preparation of reagents and methodology for measurement of sulfatides and arylsulfatase A enzymatic activity in dried blood spots.
Journal: Molecular genetics and metabolism
Published: March 29, 2025
Adult-onset metachromatic leukodystrophy: a novel genotype with a distinct phenotype.
Journal: Psychiatric genetics
Published: March 27, 2025
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
Sphingolipidoses: expanding the spectrum of α-synucleinopathies.
Journal: Journal of neural transmission (Vienna, Austria : 1996)
Published: March 06, 2025
Last Updated: 10/31/2025