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Two recent experimental treatments improve visual function in mouse models of retinitis pigmentosa

Two recent experimental treatments - one involving skin-derived induced pluripotent stem (iPS) cell grafts, the other gene therapy - have been shown to produce long-term improvement in visual function in mouse models of retinitis pigmentosa (RP), according to the Columbia University Medical Center (CUMC) scientists who led the studies. At present, there is no cure for RP, the most common form of inherited blindness.

20 Dec 2012

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

QLT expands QLT091001 Phase 1b LCA study to include patients with Retinitis Pigmentosa

QLT Inc. today announced expansion of the Phase 1b clinical proof-of-concept study of QLT091001 in the treatment of Leber Congenital Amaurosis (LCA) to include patients with Retinitis Pigmentosa (RP), a set of hereditary retinal diseases demonstrating clinical features similar to LCA.

28 Oct 2010

New gene therapy to restore retinal cone function and day vision

Veterinary ophthalmology researchers from the University of Pennsylvania have used gene therapy to restore retinal cone function and day vision in two canine models of congenital achromatopsia, also called rod monochromacy or total color blindness.

22 Apr 2010

Retinitis Pigmentosa: Embryonic stem cells for replacement of diseased retinal cells and restoration of sight

An international research team led by Columbia University Medical Center successfully used mouse embryonic stem cells to replace diseased retinal cells and restore sight in a mouse model of retinitis pigmentosa. This strategy could potentially become a new treatment for retinitis pigmentosa, a leading cause of blindness that affects approximately one in 3,000 to 4,000 people, or 1.5 million people worldwide.

25 Feb 2010

Scientists still searching for valid biomarker for schizophrenia and bipolar disorder

Schizophrenia and bipolar disorder affect tens of millions of individuals around the world. These disorders have a typical onset in the early twenties and in most cases have a chronic or recurring course.

4 Feb 2010

Acucela to present data on its ACU-4429 visual cycle modulator for dry AMD

Acucela, a clinical-stage biotechnology company focused on developing new treatments for blinding eye diseases, announced today that data for the company’s novel visual cycle modulator, ACU-4429, a potential oral treatment for dry age-related macular degeneration (dry AMD), will be featured at the 8th International Symposium on Ocular Pharmacology and Therapeutics (ISOPT) being held in Rome, Italy from December 3 to 6, 2009.

23 Nov 2009

Herbal, nutritional supplements linked to side effects ranging from dry eye to retinal hemorrhages and transient visual loss

An estimated 42 percent of Americans use herbal medicines or nutritional supplements. Many people taking these products and their physicians are unaware of the adverse reactions they can cause.

13 Oct 2004