Rare liver cancer found in toddler with Alagille syndrome
A toddler boy with cirrhosis, or permanent liver scarring, due to Alagille syndrome developed a hepatoblastoma, a type of liver cancer that typically occurs…
Margarida Maia, PhD, Science Writer — 
Articles by Margarida Maia
Autoimmune hepatitis treatment zetomipzomib trial seeks patients
A Phase 2a clinical trial testing Kezar Life Sciences’ investigational therapy zetomipzomib in adults with autoimmune hepatitis who did not respond well to standard…
New mutations tied to rapidly progressing intrahepatic cholestasis
Researchers in China have identified five new mutations in the NR1H4 gene in three boys with progressive familial intrahepatic cholestasis type 5 (PFIC5) and…
UDCA plus elafibranor best for treatment-resistant PBC: Analysis
Combining ursodeoxycholic acid (UDCA) with elafibranor, an experimental oral therapy for primary biliary cholangitis (PBC), may be the best treatment combination at lessening signs…
LB-P8 probiotic on FDA fast track for primary sclerosing cholangitis
The U.S. Food and Drug Administration (FDA) has granted fast track designation to LB-P8, LISCure Biosciences’ experimental live bacterium product that is headed toward…
Brain changes seen in children with neonatal cholestasis: Study
Brain scans have shown children with neonatal cholestasis tend to have brain abnormalities, a review study found. Bleeding was the most common, especially in…
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…
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…
