X-linked adrenoleukodystrophy (X-ALD
) affects 1 in 17,000 people worldwide.
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.
, 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.
(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
M2 PHARMA-July 21, 2017-NeuroVia Closes USD 14m Series A Financing Round; Will Fund X-ALD