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

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‐oxidation, leading to accumulation in plasma and tissues. Excess VLCFA drives impaired cellular functions (e.g. disrupted mitochondrial fun...

Descripción completa

Detalles Bibliográficos
Autores principales: Monternier, Pierre‐Axel, Singh, Jaspreet, Parasar, Parveen, Theurey, Pierre, DeWitt, Sheila, Jacques, Vincent, Klett, Eric, Kaur, Navtej, Nagaraja, Tavarekere N., Moller, David E., Hallakou‐Bozec, Sophie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2022
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9545763/
https://www.ncbi.nlm.nih.gov/pubmed/35510808
http://dx.doi.org/10.1002/jimd.12510

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9545763/
https://www.ncbi.nlm.nih.gov/pubmed/35510808
http://dx.doi.org/10.1002/jimd.12510

Ejemplares similares