Oral drug may cure a form of childhood blindness

Oral drug may cure a form of childhood blindnessToronto: A new oral medication is showing significant progress in restoring vision to patients having an inherited retinal disease which causes visual impairment ranging from reduced vision to complete blindness.

Until now, the disease called Leber Congenital Amaurosis (LCA) has been untreatable.

“This is the first time that an oral drug has improved the visual function of blind patients with LCA,” said the study’s lead author Robert Koenekoop from McGill University Health Centre (RI-MUHC) in Montreal, Canada.

“By giving patients with RPE65 or LRAT mutations an oral retinoid intermediate (QLT091001), most patients’ vision improved rapidly,” Koenekoop said.

“We discovered that a certain portion of the retinal cells that were not working because of the lack of 11-cis retinal could be woken up,” Koenekoop noted.

The study, published in The Lancet, involved 14 participants from around the world with LCA ranging in age from six to 38 years old.

Their blindness was caused by either mutations in the genes RPE65 or LRAT, leading to a serious defect in the retinoid cycle.

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