EU initiative aims to improve early detection of rare genetic diseases
An initiative in the European Union (EU) aims to speed up the diagnosis of rare genetic disorders such as Alagille syndrome in newborns using…
News
PSC may increase ulcerative colitis risk, but not Crohn’s disease
Genetic variations that predispose people toward developing primary sclerosing cholangitis (PSC) are significantly associated with a higher risk of ulcerative colitis, a primary type…
Livmarli now approved in EU to treat adults, children with PFIC
Livmarli (maralixibat), an oral medication from Mirum Pharmaceuticals, is now approved in the European Union to treat progressive familial intrahepatic cholestasis (PFIC) in…
Ferroptosis, type of cell death, may be therapeutic target for MASLD
A type of cell death called ferroptosis contributes to metabolic dysfunction-associated steatotic liver disease (MASLD), a form of fatty liver disease that occurs more…
Biliary atresia children show delays after living donor liver transplant
Children with biliary atresia show developmental problems, including language delays, following a living donor liver transplant (LDLT) in infancy and a second surgery to…
More studies support use of CM-101 antibody therapy now in PSC trial
High levels of CCL24, the protein targeted by Chemomab Therapeutics’ investigational antibody-based therapy CM-101, are associated with biological pathways contributing to the progression of…
Baby with rare PFIC type thrives after liver transplant: Case study
A living donor liver transplant successfully managed liver failure in a 6-month-old boy with progressive familial intrahepatic cholestasis type 8 (PFIC8) that was unresponsive to…
Clinical genome sequencing aids in rare genetic disease diagnosis: Study
Clinical genome sequencing, or cGS, testing to detect a rare genetic disease such as Alagille syndrome, resulted in a positive diagnosis of varying disorders…
HCV, HIV opt-out testing program to now be evaluated nationwide
A team of researchers, backed by a grant totaling more than $3 million, will study the feasibility of a nationwide opt-out testing program to detect…
Volixibat shows early promise for reducing PBC itching, fatigue: Data
Daily treatment with Mirum Pharmaceuticals’ oral candidate volixibat significantly reduced itching — medically known as pruritus — and fatigue for adults with primary…
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