New Treatment for Leber’s Congenital Amaurosis (LCA)

multicolored pills

Wouldn’t it be great if all you had to do to cure your child’s blindness was to give them some medicine? Medicine they would just have to swallow. That’s what researchers are working on for people with Leber’s Congenital Amaurosis (LCA), a congenital form of blindness.

Up until now, there has been no easy way to treat LCA, which affects about one person in 80,000. This oral medication works by “waking up” the retinal cells so they can do their job, resulting in better visual acuity and/or increased visual field in those who tried it. It’s the first time an oral medication has been developed that has actually improved the visual function of blind patients with LCA.

A study has been done involving 14 people ages 6 to 38 with LCA from around the world, each with blindness caused by either mutations in the RPE65 or LRAT genes; in each case, there was rapid improvement.

The study’s lead author is Dr. Robert Koenekoop, director of the McGill Ocular Genetics Laboratory at The Montreal Children’s Hospital of the MUHC. He’s a Professor of Human Genetics, Pediatric Surgery and Ophthalmology at McGill.

The full article can be found on the Foundation Fighting Blindness website and you can read it here.


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