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X-linked retinitis pigmentosa (XLRP) is a severe form of retinitis pigmentosa, a group of IRDs that that cause serious vision impairment and blindness. Retinitis pigmentosa affects about 1 in 3,500 people, with XLRP accounting for approximately 15% of all retinitis pigmentosa cases.

Visual impairment in XLRP initially manifests with loss of night vision during adolescence, followed by progressive constriction of the visual field in adulthood, and eventual advancement to central vision loss and legal blindness by 30 to 40 years of age. Due to its inheritance pattern, XLRP affects men and women differently, with men more likely to experience severe symptoms. One in 40,000 people in the U.S. are expected to be living with XLRP, and it’s estimated that approximately 20,000 XLRP patients are among the U.S., Japan and EU5.

XLRP is most commonly caused by a defect in the RPGR gene, which encodes a protein called X-linked retinitis pigmentosa GTPase regulator (RPGR) that plays a vital role in the development of the cells that make up the retina, a thin layer of tissue found on the back wall of the eye. Absence of this functional protein results in poorly functioning rod and cone photoreceptors, parts of the eye which are responsible for vision at low and high light levels, as well as seeing color.