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Etidronate: A Ray of Hope for a Rare Arterial Calcification Disorder

Etidronate: A Ray of Hope for a Rare Arterial Calcification Disorder
Etidronate: A Ray of Hope for a Rare Arterial Calcification Disorder

A Promising Drug for Rare Genetic Condition Causing Arterial Calcification

A recent clinical trial has shed light on a potential treatment for a rare genetic condition called arterial calcification due to deficiency of CD73 (ACDC). This condition causes excessive calcium buildup in the arteries, primarily affecting the legs and hands, leading to pain, difficulty walking, and potential limb loss.

Background and Disease Mechanism

ACDC, an extremely rare disease affecting less than 20 individuals worldwide, is caused by a genetic mutation that impairs the function of the CD73 protein. This leads to a biochemical imbalance that promotes calcium deposition in the arteries.

Clinical Trial Findings

Researchers tested an existing drug, etidronate, in a small-scale trial involving seven individuals with ACDC. The drug was administe

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