Newborn Screening for X-Linked Adrenoleukodystrophy

• Hoofdauteurs: Ann B. Moser (Auteur), Richard O. Jones (Auteur), Walter C. Hubbard (Auteur), Silvia Tortorelli (Auteur), Joseph J. Orsini (Auteur), Michele Caggana (Auteur), Beth H. Vogel (Auteur), Gerald V. Raymond (Auteur)
• Formaat: Boek
• Gepubliceerd in: MDPI AG, 2016-12-01T00:00:00Z.
• Onderwerpen: article
• Online toegang: Connect to this object online.

Early diagnosis of males with X-linked adrenoleukodystrophy (X-ALD) is essential for preventing loss of life due to adrenal insufficiency and for timely therapy of the childhood cerebral form of X-ALD with hematopoietic cell transplantation. This article describes X-ALD, the current therapies, the h...