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Archive for the Stargardt disease Category

All-Star Vision

When the Baltimore Orioles’ Adam Jones makes a great catch — a feat the Gold Glove centerfielder accomplishes on a regular basis — he isn’t thinking about his retinas. But in the five seconds it takes a baseball to leave an opponent’s bat and reach Jones’ glove, his retinas are processing an enormous amount of real-time visual information — continual changes in the contrast, velocity and trajectory of the ball as it rockets out of the infield, reaches high into the stadium lights (or the sun) and descends into the outfield.
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FFB’s New Research Playlists on Youtube

Image of Eye and YouTube IconEvery field of research has its “rock stars,” specialists so revered for their knowledge, expertise and experience, they’re famous in certain circles worldwide. And when it comes to retinal-disease research, the stars are invariably linked to the Foundation Fighting Blindness, which either funds or has funded their vision-saving work. So, after interviewing a handful of them recently, we’ve put together a few research-oriented playlists on our revamped YouTube page.
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DHA and EPA for Stargardt Disease — an Evolving Story

Dr. David KrizajI’ve been conducting or overseeing scientific research for more than 40 years (yikes!), but I continue to be humbled by its nuances, complexities and ambiguities. Sometimes, just when you think you have something figured out, you find evidence to the contrary. Sometimes, as Vladimir Lenin said, progress is made by taking “one step back, two steps forward.”
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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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Safely Harnessing the Power of Stem Cells

Image of a Stem CellStem cells are a highly promising treatment approach for a wide range of conditions and diseases, because they can be used to replace virtually any type of cell or tissue in the body lost to disease or injury. In the not-too-distant future, in fact, researchers will be able to turn stem cells into new photoreceptors for people who have lost vision to a retinal disease.

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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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Curing Blindness, Part 2: Dick’s Story

Image of Dick CoulsonTwo days ago, as part of FFB’s “Light the Way to a Cure” fundraising campaign, I shared the story of Corey Haas, as an example of clinical trial participants benefitting from gene therapy studies the Foundation supports. In his case, the therapy’s for a retinal disease called Leber congenital amaurosis. Today, I’d like to share the story of Dick Coulson (pictured, left), who has age-related macular degeneration (AMD), the leading cause of blindness in people age 55 and older. It’s also the target of two treatments that are currently in clinical, or human, trials, both of which are derived from stem cells.
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