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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Archive for the Pharmaceuticals Category
Researchers 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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“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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You 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.
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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At 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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Without a doubt, as chief research officer at FFB, I get more questions about clinical trials – also known as human studies – than any other topic. So, I thought it would be useful to provide a list of the major clinical trials underway right now for retinal disease treatments. Continue Reading…
Ryan Lochte, I didn’t get a chance to meet. I did, however, get a picture taken with Michael J. Fox, who was very cordial but focused most of his attention—and rightly so—on a little girl with muscular dystrophy. Now, actor Steve Buscemi is a character and a half. I was there with Leah Bartos, who’s Mrs. New York, and because I’m Mrs. World, and the two of us were wearing crowns and sashes, he took a look at us and, before a photo was snapped, said, “Wow, let me get in between these two ladies.”
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A couple weeks ago, I was in China, to visit with Dr. Ruifang Sui, a Foundation-funded clinician-researcher at the Peking Union Medical College Hospital in Beijing. During my time there, I felt like I had never left the United States. Don’t get me wrong — there’s no mistaking Beijing for Baltimore (the location of our national office) or Mandarin for English. But when it comes to retinal degenerations, we and the Chinese have a lot in common.
Steve is highly respected for his expertise and tireless commitment to finding treatments and cures for vision-robbing retinal diseases. 
As the Foundation's senior science writer, Ben writes science and research articles for the Foundation’s website, newsletters and Eye on the Cure blog. 
As the Foundation's senior writer, Rich writes and edits content for all of the Foundation’s print and online publications, including its blog, Eye on the Cure. 
Retinal Research Developments Go Mainstream (at The Wall Street Journal)
By Rich Shea on July 25, 2012 | 2 Comments
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