New NR1H4 mutation identified in baby girl with PFIC5
A new mutation in the NR1H4 gene was identified as the cause of progressive familial intrahepatic cholestasis type 5 (PFIC5) in a baby girl…
Marisa Wexler, MS, Senior Science Writer — 
Articles by Marisa Wexler
WHO: Despite advances in care, deaths from hepatitis increasing
Technologies to prevent, detect, and treat viral hepatitis have advanced considerably in recent decades, but the number of people dying from these infections continues…
Nonprofit funds research to test for pregnancy complication ICP
A nonprofit group has donated $60,000 to support researchers at Yale University School of Medicine working to expand access to a new test that can…
Fatty liver disease treatment DD01 gets FDA fast track designation
The U.S. Food and Drug Administration (FDA) has granted fast track designation to DD01 as a potential treatment for a severe manifestation of fatty…
Pruritus treatment Livmarli should be reimbursable: Canadian panel
A Canadian Agency for Drugs and Technologies in Health (CADTH) committee has recommended that Livmarli (maralixibat) be covered by the country’s publicly funded health…
FDA expands Vemlidy to chronic hepatitis B patients as young as 6
The U.S. Food and Drug Administration (FDA) has again expanded its approval of Gilead Sciences’ medication Vemlidy (tenofovir alafenamide) for chronic hepatitis B…
Livmarli led to ‘dramatic responses’ in PFIC trial, Mirum exec says
The Phase 3 trial that supported the U.S. approval of Livmarli (maralixibat) for progressive familial intrahepatic cholestasis (PFIC) involved the broadest range of patients,…
Brain blood vessel abnormalities in Alagille usually stable over time
Blood vessel abnormalities in the brain, such as unusually narrow or bulging vessels, are common in children with Alagille syndrome and remain generally stable over…
FDA to decide on full approval for Ocaliva in treating adults with PBC
The U.S. Food and Drug Administration (FDA) agreed to review an application seeking full and formal approval of Ocaliva (obeticholic acid) as a…
FDA approves Mirum’s Livmarli for PFIC patients ages 5 and older
The U.S. Food and Drug Administration (FDA) has approved Mirum Pharmaceuticals’ Livmarli (maralixibat) to reduce pruritus, or itching, in adults and children, 5 years…