Tauroursodeoxycholate—bile acid with chaperoning activity: molecular and cellular effects and therapeutic perspectives

M Kusaczuk - Cells, 2019 - mdpi.com
Tauroursodeoxycholic acid (TUDCA) is a naturally occurring hydrophilic bile acid that has
been used for centuries in Chinese medicine. Chemically, TUDCA is a taurine conjugate of …

Biomarker identification, safety, and efficacy of high-dose antioxidants for adrenomyeloneuropathy: a phase II pilot study

C Casasnovas, M Ruiz, A Schlüter, A Naudí… - …, 2019 - Springer
Abstract X-Adrenoleukodystrophy (X-ALD) and its adult-onset, most prevalent variant
adrenomyeloneuropathy (AMN) are caused by mutations in the peroxisomal transporter of …

Therapeutic Strategies for X-Linked Adrenoleukodystrophy, a Representative Peroxisomal Disorder

M Morita - Peroxisomes: Biogenesis, Function, and Role in …, 2019 - Springer
Abstract X-linked adrenoleukodystrophy (X-ALD) is the most frequent peroxisomal disorder,
and is caused by dysfunction of the peroxisomal ABC protein ABCD1. X-ALD patients with …

B cell depletion can be effective in multiple sclerosis but failed in a patient with advanced childhood cerebral X-linked adrenoleukodystrophy

H Rosewich, S Nessler, W Brück… - Therapeutic Advances …, 2019 - journals.sagepub.com
Rituximab exerts its clinical efficacy by its specific pattern of depletion of CD20+ B
lymphocytes and it has been demonstrated that rituximab is an effective treatment for …

Failure to clear developmental apoptosis contributes to the pathology of RNASET2-deficient leukoencephalopathy

N Hamilton, HA Rutherford, HM Isles, JJ Petts, T Weber… - bioRxiv, 2019 - biorxiv.org
The contribution of microglia in neurological disorders is emerging as a leading driver rather
than a consequence of pathology. RNAseT2-deficient leukoencephalopathy is a severe …