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Archive for the Pharmaceuticals Category

Total Blindness and Non-24 Sleep Disorder

The circadian clockRecently, you may have been hearing and seeing commercials for a blindness-related sleep disorder called Non-24. In the radio ads I’ve heard, the narrator says he’s totally blind and suffers from the condition. The media spots are sponsored by Vanda Pharmaceuticals, which recently received approval from the U.S. Food and Drug Administration for a drug called Hetlioz to treat Non-24.
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A Sight-Saving ‘Fishing Expedition’

A zebrafish, the subject of retinal researchCould 500,000 zebrafish lead to the discovery of treatments that prevent vision loss?

Jeff Mumm, Ph.D., a retinal researcher at Johns Hopkins University, thinks so. He’s devised an innovative test that uses the tiny fish for screening potential vision-saving compounds. His project is funded through the Foundation’s Translational Research Acceleration Program (TRAP), which was established in 2008 to expedite the advancement of potential retinal-disease therapies into human studies.
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The Foundation’s Center Grants Foster Essential Retinal Research Collaborations

Question: How many researchers does it take to develop a retinal-disease treatment? (No, this isn’t a politically incorrect joke.) The answer is near the end of this article, but no looking ahead!
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Patient Registries Help Advance Research for Rare Diseases

Image of Keyboard and StethiscopeRare disease research is challenging, because patient information about the conditions is inherently limited. Patient recruitment for clinical trials can be especially difficult, because so few people are affected.

To address these issues, a number of foundations, patient advocacy groups and governmental institutions have launched online registries to collect patient information for use by researchers, doctors and public health experts.
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Proving a Vision-Saving Treatment Works

Dr. Hendrik Scholl conducts an electroretinogram, or ERG, with a patient at the Wilmer Eye Institute.I am very excited about ProgSTAR, the Foundation’s new study monitoring and documenting the progress of vision loss and retinal changes in people with Stargardt disease. On the surface, the study might not sound very exciting, because it isn’t evaluating a potential cure. However, the information gleaned from ProgSTAR will be of enormous value in designing future clinical trials for Stargardt disease treatments.
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Staying Alive: Saving Retinal Cells to Preserve Vision

Woman Receives Eye dropsResearchers are developing a number of promising treatment approaches for blinding retinal diseases, which include: correcting genetic defects; replacing lost retinal cells with new ones; and implanting electronic chips, like the recently FDA-approved Argus II. But, sometimes, saving vision simply comes down to keeping retinal cells alive, or at least slowing their degeneration. Known as “neuroprotection,” this approach isn’t just for the retina — it has the potential to preserve and protect all kinds of neural cells, including brain tissue and cells of the central nervous system.
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Moving Vision-Saving Treatments Out to the People Who Need Them

A lab technician“Translational research” is the mantra for many of the retinal scientists funded by the Foundation Fighting Blindness. In a nutshell, the phrase refers to the advancement of vision-saving therapies from laboratories into clinical trials and out to the people who need them.
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Orphan Drugs Get Special Treatment

Image of lab equipmentYou wouldn’t think that being called an “orphan” is a good thing. But for developers of treatments for rare diseases — including inherited retinal conditions — “orphan” status provides valuable benefits, such as tax incentives, access to special research grants and assistance with clinical trial design. The orphan designation also gives seven years of market exclusivity to the developer of a treatment.

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Found in Translation: Advancing Treatments Into Human Studies

A clinical trial patient is examined

Photo courtesy of the National Eye Institute

I am always excited when a new research paper comes across my desk reporting on an emerging treatment that has saved or restored vision in an animal or cell-based model of retinal disease. The advancement provides meaningful hope for a therapy that can benefit people. But it raises a big question for the Foundation Fighting Blindness: What will it take to move the treatment into and through human studies?
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Top 12 Research Advancements of 2012

Image of lab beakerAt the Foundation Fighting Blindness, we are always looking forward. Our scientists are continually focused on achieving the next sight-saving breakthrough, and our donors and volunteers are always looking for new opportunities to raise more money to drive the research.

But for a moment, as we put the wraps on 2012, it is very inspiring to look back on the past year and reflect on the many exciting advancements that have been made in our quest for treatments and cures.
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