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Archive for the Pharmaceuticals Category

Need-to-Know Information about Clinical Trials

clinical trial patientWith about 15 clinical trials underway for inherited retinal diseases, and several more poised to begin in a few years, patients are eager to sign up for access to potential vision-saving therapies.
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CME – What It Is, and Why People with Retinal Diseases Should Know About It

Dr. FishmanIn this Q&A, Gerald Fishman, M.D., a world-renowned clinical researcher and longstanding member of FFB’s Scientific Advisory Board, discusses cystoid macular edema (CME), a potentially damaging accumulation of fluid in the retina affecting up to 32 percent of people with retinitis pigmentosa (RP) and related conditions. It’s a complication that can make matters worse for retinas already fragile from degenerative disease. While diagnosis isn’t always straightforward, CME can be treated to minimize vision loss.
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The Top Research Advancements of 2014: How Fast Can We Go?

lab photoAs we approach 2015, it’s inspiring to look back on 2014 and recount the numerous advancements we’ve made in developing vision-saving treatments and cures. When I joined the Foundation nearly a decade ago, virtually nothing was in a human study. We were curing lots of blind mice, and clinical trials seemed elusive. But, today, more than a dozen promising therapies are being evaluated in people, and at least a dozen more clinical trials are expected to begin in the next few years.
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A Spice for the Eyes

TumericTurmeric, also known as “the golden spice,” has been around for more than 4,000 years. In India, where Radha Ayyagari, Ph.D., grew up, it’s used widely as both a food spice and an herbal medicine for treating a variety of diseases, including cancer, arthritis, urinary-tract infections and digestive disorders. It has been scientifically established that curcumin, the active ingredient in turmeric, is a potent antioxidant. So it’s perhaps no surprise that Dr. Ayyagari, a professor of ophthalmology at the University of California, San Diego, is developing an extract from turmeric that may be useful in slowing retinal degeneration.
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Low-Hanging Fruit: Repurposing Drugs to Treat Retinal Diseases

Low-hanging fruitMany years ago, while picking apples with my uncle, he advised me to “pick the low-hanging fruit.” That way, he told me, you can fill your basket faster and not be as tired at the end of the day. I think the same can be said of drug repurposing—the process of evaluating a drug prescribed for one disease to see if it can safely and effectively treat another disease.
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ARVO 2014: European Collaboration Developing Cross-Cutting, Vision-Saving Therapies

The DRUGSFORD collaborativeOne of the most intriguing reports on therapy development at the annual meeting of the Association for Research in Vision and Ophthalmology came from a European collaborative project called DRUGSFORD, which is derived from the phrase “drugs for retinal degeneration.” With funding from the European Union, the project brings together three research groups and two companies to develop a therapy that can treat a disease pathway common to many retinal degenerations. Simply put, they’re creating therapies that can save vision in as many people as possible, independent of the genetic cause of disease.
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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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