Results 111 to 120 of about 4,323 (173)

A molecular journey on the pathogenesis of primary hyperoxaluria. [PDF]

Curr Opin Nephrol Hypertens
Cellini B.
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]

Clin Pharmacokinet
Zhang S, Gamallo P, Rawson V.
europepmc   +1 more source

Preclinical evaluation of AGT mRNA replacement therapy for primary hyperoxaluria type I disease. [PDF]

Sci Adv
Yang T, Ge J, Huang L, Zhu X, Zhang D, Tang S, Zhao J, Ma Y, Long M, Bo X, Li J, Zhang Y, Yuan Q, Sharma AD, Ott M, Geng H, Zhao Y, Zhang L, Shen H, Li H, Li D, Wan P, Xia Q.   +22 more
europepmc   +1 more source

Functional analysis of amino acid substitutions within human AGT1 in a cell-based platform to support the diagnosis of primary hyperoxaluria type 1. [PDF]

J Biol Chem
Gatticchi L, Bellezza I, Rumsby G, Glover M, Cellini B.   +4 more
europepmc   +1 more source

Primary hyperoxaluria type I diagnosed after a kidney transplant presenting with subcutaneous calcification: a case report of sodium thiosulfate treatment. [PDF]

Front Pharmacol
Wu M, Lu J, Wang YJ, Li YQ, Wei Q, Gong YX, Tang RN.   +6 more
europepmc   +1 more source

Primary hyperoxaluria type 3: from infancy to adulthood in a genetically unique cohort. [PDF]

Pediatr Nephrol
Julius M, Lavsky HS, Kalfon L, Kfir NC, Herskovits M, Wiesmann I, Zaccai TCF.   +6 more
europepmc   +1 more source

Unveiling primary Hyperoxaluria type 1: a fortuitous discovery through bone marrow biopsy. [PDF]

Oxf Med Case Reports
Aaboudech TY, Znati K, Jahid A, Sassi S, Driouich S, Zouaidia F, Bernoussi Z.   +6 more
europepmc   +1 more source

The efficacy and safety of RNA interference for the treatment of primary hyperoxaluria: a systematic review and meta-analysis. [PDF]

Clin Kidney J
Yu H, Zhong H, Liu Y, Zhang G.
europepmc   +1 more source

Cutting through the stones: Unlocking therapeutic potential with gene editing tools for primary hyperoxaluria type 1. [PDF]

Mol Ther
Schneller J, Du W, Ding H.
europepmc   +1 more source