Sodium 4-phenylbutyrate delayed transplant need in 2 PFIC2 children
Treatment with an oral medication called sodium 4-phenylbutyrate — approved to treat diseases in which the body fails to remove a waste called ammonia…
Margarida Maia, PhD, Science Writer—
Articles by Margarida Maia
Bortezomib helps girl avoid liver transplant rejection, return of PFIC2
Bortezomib, an anticancer medication, prevented a recurrence of progressive familial intrahepatic cholestasis type 2 (PFIC2) after a liver transplant by helping a young girl…
Livmarli eases itch for broader pediatric population with Alagille
Livmarli (maralixibat) safely and effectively eases itching caused by Alagille syndrome in children who would have been excluded from the clinical trials that…
New JAG1 mutations seen as likely cause of Alagille in 2 young boys
Researchers have identified two new mutations in JAG1, a gene linked to Alagille syndrome, that each resulted in a unique clinical profile for two…