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Childhood cerebral adrenoleukodystrophy (CCALD) in France: epidemiology, natural history, and burden of disease - A population-based study

BACKGROUND: X-linked adrenoleukodystrophy (ALD) is a rare metabolic and neurodegenerative disorder belonging to the group of leukodystrophies, with an estimated incidence around 1:25 000 newborns worldwide, mostly among men. Childhood Cerebral ALD (CCALD) is the most severe form with a poor prognosi...

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Detalles Bibliográficos
Autores principales:	Sevin, Caroline, Hatteb, Samira, Clément, Aurore, Bignami, Fabrizia, Chillotti, Louis, Bugnard, Françoise, Bénard, Stève, Boespflug-Tanguy, Odile
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2023
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10416383/ https://www.ncbi.nlm.nih.gov/pubmed/37563635 http://dx.doi.org/10.1186/s13023-023-02843-x

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