Newborn Screening for X-Linked Adrenoleukodystrophy

• Main Authors: Ann B. Moser (Author), Richard O. Jones (Author), Walter C. Hubbard (Author), Silvia Tortorelli (Author), Joseph J. Orsini (Author), Michele Caggana (Author), Beth H. Vogel (Author), Gerald V. Raymond (Author)
• Format: Book
• Published: MDPI AG, 2016-12-01T00:00:00Z.
• Subjects: article
• Online Access: 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...