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

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

Abstract 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...

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Bibliographic Details
Main Authors: Caroline Sevin, Samira Hatteb, Aurore Clément, Fabrizia Bignami, Louis Chillotti, Françoise Bugnard, Stève Bénard, Odile Boespflug-Tanguy
Format: Article
Language:English
Published: BMC 2023-08-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Adrenoleukodystrophy
CCALD
Epidemiology
Claims
Registry
HSCT
Online Access:https://doi.org/10.1186/s13023-023-02843-x

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