Primary hyperoxaluria type 1—current practice in the siRNA era: an ERA Genes & Kidney Working Group survey
Malte P. Bartram, Giovambattista Capasso, Émilie Cornec-Le Gall, Lisa J. Deesker et autres
Background: Primary hyperoxaluria type 1 (PH1) is a rare inherited metabolic disorder leading to the formation of kidney stones, nephrocalcinosis, and kidney failure. Besides, PH1 poses the risk of developing systemic oxalosis, a life-threatening condition with oxalate deposits in multiple organ systems. …
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