N-Acetyl-l-Leucine and Neurodegenerative Disease

N-Acetyl-l-Leucine and Neurodegenerative Disease

This editorial describes the science behind a study of N -acetyl- l -leucine to treat a specific lysosomal storage disorder, Niemann–Pick disease type C.

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Bibliographische Detailangaben
Veröffentlicht in:The New England journal of medicine 2024-02, Vol.390 (5), p.467-470
1. Verfasser: Tifft, Cynthia J.
Format: Artikel
Sprache:eng
Schlagworte:
Allergy
Ataxia
Autophagy
Cell death
Childhood Diseases
Disease
Enzymes
Gene therapy
Genetics
Humans
Immunology
Inflammatory Disease
Leucine
Lysosomal storage diseases
Metabolism
Metabolites
Nervous system
Neurodegenerative Diseases
Neurology
Neuroscience
Neurosurgery
Neurosurgery General
Niemann-Pick disease
Pediatrics
Pediatrics General
Traumatic brain injury
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Beschreibung
Zusammenfassung:This editorial describes the science behind a study of N -acetyl- l -leucine to treat a specific lysosomal storage disorder, Niemann–Pick disease type C.
ISSN:0028-4793
1533-4406
DOI:10.1056/NEJMe2313791