In Case You Missed…Advancing Science in the Treatment of X-linked Retinitis Pigmentosa

Author(s)/Faculty: Byron L. Lam, MD; Lesley Everett, MD, PhD; M. Dominik Fischer, MD, PhD, FEBO
Release Date: 2/1/2022Expiration Date: 1/31/2023
Credit Type: CME / CME with ABO Continuing Certification Number of Credits: 1
Content Type: LectureProvider:
Gene therapy for inherited retinal disease (IRD) became a reality in 2017, with the approval of voretigene neparvovec-rzyl to treat Leber congenital amaurosis. Although there have been no subsequent approvals to date, numerous treatment candidates are in development for various IRDs. The most common IRD is retinitis pigmentosa (RP), which is caused by inherited or mutated genes. Therefore, developing replacement-based gene therapies for all genetic forms of RP is challenging. X-linked RP (XLRP) causes blindness in men, with the phenotype being one of the most severe among all causes of RP and is characteristic for its early onset and rapid progression to blindness. With advances in the use of gene therapy being published or presented on a frequent basis, retina specialists and other health care providers are challenged to remain current regarding this information and can lack knowledge about emerging gene therapies that may transform the XLRP treatment paradigm. In this In Case You Missed activity, featuring commentary on abstracts presented at recent conferences, expert clinicians discuss the latest updates in XLRP diagnosis, epidemiology, pathophysiology, and treatment.