Custom Antisense Oligonucleotide (ASO) Therapy for Retinal Disease in One Patient

Retinitis pigmentosa (RP) is a degenerative eye disease that impacts about 100,000 individuals in the U.S. Often diagnosed in early adulthood, but sometimes presenting in teenagers or even younger children, RP progressively narrows the visual field, impairs night vision and can ultimately lead to blindness. But there’s new hope for some patients with RP with the recent development of a novel antisense oligonucleotide (ASO) drug therapy, spearheaded by Children’s Hospital Colorado and the University of Colorado School of Medicine.
A decade ago, Emily McCourt, MD, from Children’s Colorado’s Department of Pediatric Ophthalmology, diagnosed a toddler with posterior column ataxia with retinitis pigmentosa, or PCARP, a pivotal moment that set the course for pioneering work in personalized treatment. In this video, Dr. McCourt discusses the process of creating a new precision medicine drug through an N-of-one trial, obtaining FDA approval, and creating a pipeline to give more patients the opportunity for targeted therapies.
For more information about retinitis pigmentosa, please visit: www.childrenscolorado.org/adv...
Connect with Children’s Colorado:
Instagram: / childrenscolo
Facebook: / childrenshospitalcolorado
Twitter: / childrensco_pro
LinkedIn: / children's-hospital-co...
Pinterest: / childrenscolo
Website: www.childrenscolorado.org