Skin fibroblasts of patients with geleophysic dysplasia due to FBN1 mutations have lysosomal inclusions and losartan improves their microfibril deposition defect.
Pasquale PiccoloValeria SabatinoPratibha MithbaokarElena PolishchukJohn HicksRoman PolishchukCarlos A BacinoNicola Brunetti-PierriPublished in: Molecular genetics & genomic medicine (2019)
Losartan is a promising candidate drug for treatment of GPHYSD due to FBN1 defects.