X-ALD

AcronymDefinition
X-ALDX-Linked Adrenoleukodystrophy (disease)
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X-ALD is a monogenic disease, with an incidence ranging from 1:20000 to 1:100000, which is caused by mutations in the ABCD1 (ATP binding cassette subfamily D member 1) gene, located on Xq28.
In X-ALD, since cholesterol is entrapped as esters in the lamellar inclusions, it cannot be normally metabolized into T.
Pentoxifylline, a methylxanthine inhibiting the activity of tumor necrosis factor [alpha] (TNF[alpha]), an important component in the X-ALD inflammatory response, has been recently used in the treatment of X-ALD.
Do you remember the 1992 motion picture Lorenzo's Oil, which starred Nick Nolte and Susan Sarandon, about a boy who develops the rare and often deadly disease X-ALD? Fourteen years after the release of that film, Dr.
As the accumulation of VLCFAs is believed to contribute to the underlying pathology of X-ALD, these data provide additional support for the role of selective thyroid receptor beta (TRb) activation as a potential therapeutic approach to the disease.
(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.
Fibroblasts from healthy controls and patients with X-ALD were cultured in the absence or presence of 20 [micro]mol/L [D.sub.3]-C24:0 in HAM-F10 tissue culture medium supplemented with 100 mL/L fetal calf serum, penicillin (100 IU/mL), streptomycin (100 IU/mL), and glutamine (2 mmol/L).
With no curative treatment currently available, there exists an unmet medical need for a therapeutic benefit for X-ALD patients.
Evaluation of VK0214 in an in vivo model of X-linked adrenoleukodystrophy (X-ALD) recently completed.
M2 PHARMA-July 21, 2017-NeuroVia Closes USD 14m Series A Financing Round; Will Fund X-ALD Disease Treatment
Viking Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, today announced the presentation of positive data from a proof-of-concept study of VK0214 in a mouse model of X-linked adrenoleukodystrophy (X-ALD).