Multi-omics Study in Citrin Deficiency
Citrin deficiency (CD) is an underdiagnosed and understudied condition characterized by
several distinct phenotypes: 1) neonatal intrahepatic cholestasis caused by citrin
deficiency (NICCD), 2) the adaptation or silent period, 3) "failure to thrive and
dyslipidemia" ...
Age: 0 - 100 years
Gender: All
Multi-omics Study in Citrin Deficiency
Citrin deficiency (CD) is an underdiagnosed and understudied condition characterized by
several distinct phenotypes: 1) neonatal intrahepatic cholestasis caused by citrin
deficiency (NICCD), 2) the adaptation or silent period, 3) "failure to thrive and
dyslipidemia" ...
Age: 0 - 100 years
Gender: All
Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC
Open Label Extension Study to evaluate long term safety and persistence of effect of A4250 in
children with PFIC.
Age: Birth - 100 years
Gender: All