Lumasiran at birth changes the trajectory of primary hyperoxaluria type 1: same disease, different outcomes in two affected siblings.

ABSTRACT: Lumasiran, an RNA interference therapeutic, demonstrated effectiveness in clinical trials, leading to approval for primary hyperoxaluria type 1 management in all age groups. To date, little is known about its use in newborns. This study assesses, for the first time, the oxalate and glycolate metabolism in a newborn affected by primary hyperoxaluria type 1 treated at birth. His older brother, also affected by primary hyperoxaluria type 1, experienced severe disease progression and significant comorbidities. These challenges informed the decision to initiate immediate treatment for the younger sibling. The child was treated at 6 h of life with lumasiran 6 mg/kg subcutaneously, in combination with pyridoxin 10 mg/kg/day. Lumasiran 6 mg/kg was repeated at 30 and 60 days, then was reduced to 3 

SUBMITTER: Peruzzi L 

PROVIDER: S-EPMC12630153 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature