Gene Therapy Improves Functional Measures in XLRP
XLRP
Disease Category: x-linked
Patient Population: 20,000 (US & Europe)
Active Clinical Trials: 17
Treatment Options: Nothing approved
Strategies to Preserve Eye Health: Protection from UV exposure; vitamin supplements, in some cases
Institution(s) Conducting Research: University of Texas, Houston, Radboud University, University of Alberta, Casey Eye Institute
A FACE OF RP
TBD
Brooklyn, NY
BRIEF DESCRIPTION
X-linked retinitis pigmentosa (XLRP) is a rare, inherited genetic eye disease that causes gradual vision loss and eventually blindness. It's caused by a mutation in the RPGR gene on the X chromosome, which affects men and women differently.
XLRP is a severe form of retinitis pigmentosa (RP), a group of rare genetic eye diseases that damage light-sensitive cells in the retina. The most common type of RP is autosomal recessive, which affects about 50–60% of people with RP.