Lumasiran at birth changes the trajectory of primary hyperoxaluria type 1: same disease, different outcomes in two affected siblings. [PDF]
Peruzzi L +2 more
europepmc +1 more source
Effect of the allelic background on the phenotype of primary hyperoxaluria type I. [PDF]
Mandrile G, Cellini B, Ferraro PM.
europepmc +1 more source
Assessment of hypervolemia by cyclic 3 ` 5 `-guanosine monophosphate in pediatric patients on hemodialysis [PDF]
Gerzer, Rupert +6 more
core +1 more source
A Minor Haplotype Variant Determines the Pathogenicity of the p.Ile279Thr Substitution in the Primary Hyperoxaluria Type 1 Gene, AGXT. [PDF]
Ruta L +7 more
europepmc +1 more source
Variable treatment response to lumasiran in pediatric patients with primary hyperoxaluria type 1. [PDF]
Saffe S +8 more
europepmc +1 more source
PHYOX3: Nedosiran Long-Term Safety and Efficacy in Patients With Primary Hyperoxaluria Type 1. [PDF]
Lieske JC +10 more
europepmc +1 more source
Effective Newborn Screening for Type 1 and 3 Primary Hyperoxaluria. [PDF]
Hoppe B +10 more
europepmc +1 more source
Primary hyperoxaluria type 1 diagnosis in adult dialysis patients: prediction model assessment in a group of Italian patients. [PDF]
Ferraro PM +16 more
europepmc +1 more source
Population Pharmacokinetic and Pharmacodynamic Modelling and Simulation for Nedosiran Clinical Development and Dose Guidance in Pediatric Patients with Primary Hyperoxaluria Type 1. [PDF]
Zhang S, Gamallo P, Rawson V.
europepmc +1 more source

