Retinal Dystrophy News and Research

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EMA grants QLT positive opinions for Orphan Drug Designation of QLT091001 to treat inherited blindness

QLT Inc. today announced that QLT091001, an oral synthetic retinoid, has received positive opinions for two distinct Orphan Drug Designations by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) for the treatment of the inherited retinal degenerative diseases, Leber Congenital Amaurosis (LCA) and Retinitis Pigmentosa (RP).

10 Feb 2011

QLT's oral synthetic retinoid granted orphan drug designation for treatment of Leber Congenital Amaurosis

QLT Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for QLT091001, its oral synthetic retinoid, for the treatment of Leber Congenital Amaurosis (LCA). LCA is an inherited progressive retinal degenerative disease that leads to retinal dysfunction and visual impairment beginning at birth. QLT091001 is an orally administered synthetic retinoid replacement for 11-cis-retinal, which is a key biochemical component of the visual retinoid cycle.

8 Dec 2010

Eye implant restores some vision in retinitis pigmentosa

According to a new scientific breakthrough, a device - a tiny, three-by-three millimetre microchip, has allowed patients with inherited blindness to detect objects with their eyes, unlike a rival approach that uses an external camera. The procedure details are outlined in the journal Proceedings of the Royal Society B.

3 Nov 2010

New study holds promise for patients with LCA

A new study led by Dr. Robert Koenekoop at the Montreal Children's Hospital at the McGill University Health Centre holds promise for certain patients with Leber congenital amaurosis, an inherited retinal degenerative disease that causes significant vision loss from birth. The study, sponsored by Canadian company QLT Inc., is designed to treat patients with LCA due to genetic mutations in either retinal pigment epithelium protein 65 or lecithin:retinol acyltransferase.

27 May 2010

Interim results from QLT's Phase 1b study of QLT091001 for Leber congenital amaurosis

QLT Inc. today announced interim results from the first 3 subjects enrolled in a Phase 1b clinical proof-of-concept study of QLT091001 in the treatment of Leber congenital amaurosis (LCA), an inherited progressive retinal degenerative disease that leads to retinal dysfunction and significant visual impairment beginning at birth. QLT091001 is an orally administered synthetic retinoid replacement for 11-cis-retinal, which is a key biochemical component of visual function.

20 Apr 2010

LCA: Few possess genetic mutation necessary for positive outcome from gene therapy

Leber congenital amaurosis (LCA) is a congenital retinal dystrophy present in approximately 1 of 80,000 births. It is estimated that about 3,000 people in the United States are living with LCA and will likely become blind in their lifetimes. Recently, there has been progress in gene therapy for this condition.

7 Apr 2010

Gene therapy for retinitis pigmentosa

An international team of scientists and clinicians from the United States and Saudi Arabia are working to develop gene therapy for treating a rare, hereditary retinal disease.

31 Jul 2009

Bionic eyes from Stanford

Stanford physicists and eye doctors have teamed up to design a "bionic eye," of sorts. On Feb. 22 in the Journal of Neural Engineering, Daniel Palanker, Alexander Vankov and Phil Huie from the Department of Ophthalmology and the Hansen Experimental Physics Laboratory and Stephen Baccus from the Department of Neurobiology published a design of an optoelectronic retinal prosthesis system that can stimulate the retina with resolution corresponding to a visual acuity of 20/80

4 Apr 2005