Systematic study of ophthalmological findings in 10 patients with PEX1 -mediated Zellweger spectrum disorder.
Jessica S KaruntuFemke C C KlouwerMarc EngelenCamiel J F BoonPublished in: Ophthalmic genetics (2024)
This study highlights the ophthalmological phenotype resembling RP with moderate to severe visual impairment in patients with mild ZSD. These findings can aid ophthalmologists in diagnosing, counselling, and managing patients with mild ZSD.