ATP-binding cassette (ABC) transporters constitute one of the largest superfamilies of
conserved proteins from bacteria to mammals. In humans, three members of this family are …

Abstract X-linked adrenoleukodystrophy (X-ALD) is an inherited neurodegenerative disorder
associated with mutations of the ABCD1 gene that encodes a peroxisomal transmembrane …

The blood–brain barrier is a major impediment for targeted central nervous system (CNS)
therapeutics, especially with carboxylic acid-containing drugs. Nuclear receptor modulators …

Krabbe disease (KD) is a rare inherited demyelinating disorder caused by a deficiency in the
lysosomal enzyme galactosylceramide (GalCer) β-galactosidase. Most patients with KD …

Oligodendrocytes wrap multiple lamellae of their membrane, myelin, around axons of the
central nervous system (CNS), to improve impulse conduction. Myelin synthesis is …

With aging populations in many countries, the prevalence of neurodegenerative diseases is
expected to increase in the upcoming decades. Currently, no disease modifying therapies …

Oligodendrocytes wrap multiple lamellae of their membrane, myelin, around axons of the
central nervous system (CNS), to improve impulse conduction. Myelin synthesis is …

The blood-brain barrier is a major impediment for targeted central nervous system (CNS)
therapeutics, especially with carboxylic acid-containing drugs. Nuclear receptors …

Sommario L'adrenoleucodistrofia/adrenomieloneuropatia (ALD/AMN) è una patologia
invalidante che, ad oggi, non ha una terapia definitiva, anche se il trapianto di midollo e la …