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,…
Cholestasis
Worse outcomes risk rises with liver ailment ICP, gestational diabetes
Having gestational diabetes increases the risk of adverse outcomes in pregnancy for women with intrahepatic cholestasis of pregnancy (ICP), the most common pregnancy-related liver…
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…
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…
Probiotics may help women with ICP, but risks not yet known
Women with intrahepatic cholestasis of pregnancy (ICP) show lesser diversity across their gut bacteria than do other pregnant women, a study reports. Its findings…
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…
Protein’s levels in placenta may be key for diagnosing, treating ICP
Intrahepatic cholestasis of pregnancy (ICP), a liver disorder that can affect a pregnant woman, leads to abnormally high levels of a protein called ERp29…
Algorithm for genetic tests helps spot cause of newborn cholestasis
In infants with suspected cholestasis, targeted single-gene testing for those with symptoms that indicate a particular, associated disease and broader gene panel testing for…
New study finds PFIC3 marked by increased inflammation in liver
In a wide-ranging analysis of thousands of different proteins from liver samples from transplant patients, a team of scientists in Spain discovered potential links to…
Albireo Plans Phase 3 Trial of A4250 to Treat Children with Life-threatening Liver Disease
Children with progressive familial intrahepatic cholestasis will be able to take part in a Phase 3 clinical trial of A4250’s ability to combat the rare genetic…
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