The role of oxidative stress and inflammation in X-link adrenoleukodystrophy

J Yu, T Chen, X Guo, MI Zafar, H Li, Z Wang… - Frontiers in …, 2022 - frontiersin.org
X-linked adrenoleukodystrophy (X-ALD) is an inherited disease caused by a mutation in the
ABCD1 gene encoding a peroxisomal transmembrane protein. It is characterized by the …

Activating cannabinoid receptor 2 preserves axonal health through GSK-3β/NRF2 axis in adrenoleukodystrophy

J Parameswaran, L Goicoechea, L Planas-Serra… - Acta …, 2022 - Springer
Aberrant endocannabinoid signaling accompanies several neurodegenerative disorders,
including multiple sclerosis. Here, we report altered endocannabinoid signaling in X-linked …

Modulation of mitochondrial and inflammatory homeostasis through RIP140 is neuroprotective in an adrenoleukodystrophy mouse model

P Ranea‐Robles, J Galino, L Espinosa… - Neuropathology and …, 2022 - Wiley Online Library
Aims Mitochondrial dysfunction and inflammation are at the core of axonal degeneration in
several multifactorial neurodegenerative diseases, including multiple sclerosis, Alzheimer's …

Therapeutic potential of deuterium‐stabilized (R)‐pioglitazone—PXL065—for X‐linked adrenoleukodystrophy

PA Monternier, J Singh, P Parasar… - Journal of inherited …, 2022 - Wiley Online Library
X‐linked adrenoleukodystrophy (ALD) results from ABCD1 gene mutations which impair
Very Long Chain Fatty Acids (VLCFA; C26: 0 and C24: 0) peroxisomal import and β …

Translational and clinical pharmacology considerations in drug repurposing for X‐linked adrenoleukodystrophy—A rare peroxisomal disorder

JH Tieu, SA Sahasrabudhe, PJ Orchard… - British Journal of …, 2022 - Wiley Online Library
X‐linked adrenoleukodystrophy (X‐ALD) is an inherited, neurodegenerative rare disease
that can result in devastating symptoms of blindness, gait disturbances and spastic …

Beneficial effects of the direct AMP-kinase activator PXL770 in in vitro and in vivo models of X-linked adrenoleukodystrophy

PA Monternier, P Parasar, P Theurey, PG Dagorn… - … of Pharmacology and …, 2022 - ASPET
X-linked adrenoleukodystrophy (ALD) is a severe orphan disease caused by mutations in
the peroxisomal ABCD1 transporter gene, leading to toxic accumulation of Very Long-Chain …

The clinical spectrum of X-linked adrenoleukodystrophy: from Addison's-only in men to middle-age neurologic manifestations in women

L Fadiga, M Melo, J Saraiva, I Paiva - Hormones, 2022 - Springer
Abstract X-linked adrenoleukodystrophy (X-ALD) is caused by a mutation in the ABCD1
gene which encodes for a peroxisomal very long-chain fatty acid (VLCFA) transporter …

Pharmaceutical compositions comprising the PPAR agonist INT-131 and Nrf2 activators

BC Kahrs - US Patent 11,484,530, 2022 - Google Patents
The invention relates to pharmaceutical compositions comprising PPAR agonists and Nrf2
activators and methods of using combinations of PPAR agonists and Nrf2 activators for …