Tests of a new oral medication show the drug can improve vision in children with an inherited disease that can cause complete blindness and is currently untreatable.
“This is the first time that an oral drug has improved the visual function of blind patients with LCA (Leber congenital amaurosis),” says Robert Koenekoop, professor of human genetics, pediatric surgery, and ophthalmology at McGill University. “It is giving hope to many patients who suffer from this devastating retinal degeneration.”
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“Contrary to what was previously thought, children with LCA and defects in RPE65 or LRAT are not born with dead retinal cells; the cells can simply go dormant, and they can remain dormant for years before they eventually die. The oral drug we tested awakened these cells and allowed patients to see.”
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