ESPE Yearbook of Paediatric Endocrinology

ESPE Yearbook of Paediatric Endocrinology

ISSN 1662-4009 (online)

Published by BioScientifica
Previous issue | Volume 19 | ESPEYB19 | Next issue

Yearbook of Paediatric Endocrinology 2022

8. Adrenals

New Hope

Previous section
Clinical Trials – New Treatments
Table of contents
Next section
New Concerns

ey0019.8-10 | New Hope | ESPEYB19

8.10. The brain penetrant PPAR[gamma] agonist leriglitazone restores multiple altered pathways in models of X-linked adrenoleukodystrophy

L Rodriguez-Pascau , A Vilalta , M Cerrada , E Traver , S Forss-Petter , I Weinhofer , J Bauer , S Kemp , G Pina , S Pascual , U Meya , PL Musolino , J Berger , M Martinell , P Pizcueta

Sci Transl Med. 2021; 13(596): eabc0555. PMID: 34078742https://pubmed.ncbi.nlm.nih.gov/34078742/Brief Summary: These in vitro and in vivo studies show that the brain penetrant PPARγ agonist leriglitazone restores multiple biological pathways relevant for neuroinflammatory and neurodegenerative diseases, and particularly for X-linked adreno-leukodystrophy (X-AL...
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ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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