The PROPHECI trial: a phase II, double-blind, placebo-controlled, randomized clinical trial for the treatment of pseudoxanthoma elasticum with oral pyrophosphate

The PROPHECI trial: a phase II, double-blind, placebo-controlled, randomized clinical trial for the treatment of pseudoxanthoma elasticum with oral pyrophosphate

Abstract Background /aims. Pseudoxanthoma Elasticum (PXE, OMIM 264800) is an autosomal, recessive, metabolic disorder characterized by progressive ectopic calcification in the skin, the vasculature and Bruch’s membrane. Variants in the ABCC6 gene are associated with low plasma pyrophosphate (PPi) co...

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Main Authors: Laetitia Clotaire, Isabelle Rubera, Christophe Duranton, Jocelyn Gal, Emmanuel Chamorey, Hélène Humeau, Samir Yamani, Christine Chiaverini, Serge Willoteaux, Bernard Padovani, Laurie Mourozeau, Adam Mainguy, Stéphanie Baillif, Ludovic Martin, Georges Leftheriotis
Format: Article
Language:English
Published: BMC 2025-01-01
Series:Trials
Subjects:
Double-blind randomized clinical trial
Pyrophosphate supplementation
Arterial calcification
Pseudoxanthoma elasticum
Online Access:https://doi.org/10.1186/s13063-024-08666-w
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