A Phase 1 Study of Oral Vitamin D 3 in Boys and Young Men With X-Linked Adrenoleukodystrophy.
Keith P Van HarenKristen CunananAvni AwaniMeng GuDalia PeñaLindsay C ChromikMichal PovažanNicole C RossiJordan GoodmanVandana SundaramJennifer WinterbottomGerald V RaymondTina CowanGregory M EnnsEmmanuelle WaubantLawrence SteinmanPeter B BarkerDaniel SpielmanAli FatemiPublished in: Neurology. Genetics (2023)
This study provides Class IV evidence that fixed or weight-stratified vitamin D supplementation achieved target levels of 25-hydroxyvitamin D in boys and young men with X-ALD without brain lesions.