Homozygous novel truncating variant of CLPP associated with severe Perrault syndrome.
Rabia FaridiPamela StrattonNoemi SalmeriRobert J MorellAsma Ali KhanMuhammad A UsmaniWilliam G NewmanSheikh RiazuddinThomas B FriedmanPublished in: Clinical genetics (2024)
A female proband and her affected niece are homozygous for a novel frameshift variant of CLPP. The proband was diagnosed with severe Perrault syndrome encompassing hearing loss, primary ovarian insufficiency, abnormal brain white matter and developmental delay.