Streamlined determination of lysophosphatidylcholines in dried blood spots for newborn screening of X-linked adrenoleukodystrophy
The other entity with predominant periventricular pattern of involvement is X-linked adrenoleukodystrophy
, but the condition has posterior periventricular involvement and shows enhancing active demyelinating zone on contrast.
NASDAQ: VKTX) has released positive results from a 25-week proof-of-concept study of VK0214 in an in vivo model of X-linked adrenoleukodystrophy
(X-ALD), the company said.
Evaluation of VK0214 in an in vivo model of X-linked adrenoleukodystrophy
(X-ALD) recently completed.
Furthermore, mucopolysaccharidosis type I (MPS-I) and X-linked adrenoleukodystrophy
(X-ALD) have recently been approved by the US Secretary of Health and Human Services to be added to the RUSP (February 2016).
(X-ALD) is a rare, genetic disorder affecting 1 in 17,000 people worldwide that causes adrenal gland dysfunction and loss of the protective sheath that surrounds nerve fibers in the central nervous system.
Peroxisomal disorders can be categorized as (a) single peroxisomal enzyme deficiencies, including X-linked adrenoleukodystrophy
(X-ALD) and disorders attributable to defects in one of the peroxisomal (3-oxidation enzymes, such as acyl-CoA oxidase (AOX) deficiency and bifunctional protein (DBP) deficiency; and (b) disorders attributable to defects in peroxisome biogenesis.