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Posts tagged lca

For Rare Disease Day – The Many Benefits of Genetic Research

Rare Disease Day logoIt’s apropos that Rare Disease Day 2016 will be held on the rarest day on the calendar—Leap Day, February 29.

However, collectively, rare diseases are not uncommon. About 30 million Americans, nearly 10 percent of our population, are affected by one of 7,000 rare diseases. They’re an important public health issue, making it incumbent upon us to work hard to eradicate them.
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2015 Top 10 Retinal-Research Advances

Researcher in a labThe Foundation Fighting Blindness’ scientists, donors and volunteers made 2015 an outstanding year in our fight against blindness. As I tabulated the year’s top 10 research advances—all made possible through FFB funding—I realized that eight are for clinical trials of emerging therapies that are launching or underway.
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The Gift of Sight – Matched!

Your donation doubledIt’s holiday time, the giving season, when people not only buy gifts for family, friends and co-workers, but also donate to worthy causes—of which there are many. But, if you’ll permit me, I’d like to make a pitch for the Foundation Fighting Blindness (FFB), which happens to be offering a Holiday Board Match, meaning every donation is doubled.
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A Leap Forward: Spark Therapeutics Seeks FDA Approval for its Vision-Restoring Gene Therapy

Gene therapy recipient Yannick DuweWe’re approaching a critical milestone in the fight against blinding retinal diseases, and it has the potential to tremendously boost and accelerate the advancement of virtually all gene therapies in development for dozens of inherited retinal diseases.

Sometime in 2016, Spark Therapeutics will request marketing approval from the U.S. Food and Drug Administration (FDA) for its landmark gene therapy for retinal conditions caused by mutations in the gene RPE65, namely certain forms of Leber congenital amaurosis and retinitis pigmentosa.
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Gene Therapy Strengthens the Retina’s Connection to the Brain

diagram of brain affected by LCA therapyWhile we often think of the retina as that magical piece of tissue lining the back of the eye that makes vision possible, the brain is also an essential partner in the visual process. When light comes into the eye and is converted to electrical signals, those signals are sent through the optic nerve to the back of the brain, where they are transformed into the images we see. When children are born, pathways between the retina and the brain are in place, and, with increased interaction with the world, they become stronger over time.
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VISIONS 2015 – Dr. Shannon Boye Receives FFB Award for Excellence in Gene-Therapy Research

Shannon Boye accepting the awardWhen it comes to developing innovative gene therapies for retinal diseases, few researchers are doing more than Shannon Boye, Ph.D., and her laboratory staff of 10 at the University of Florida. In addition to advancing a gene therapy for Leber congenital amaurosis (LCA) caused by GUCY2D mutations toward a human study, Dr. Boye and her team are enhancing gene-delivery systems by making them safer to administer and able to carry larger payloads than current systems.
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Promising Research Highlighted at Meeting of Retinal Gene Therapy Experts

genesWhen it comes to fighting blinding retinal diseases, nothing has been more exciting than the advancement of gene therapies into clinical trials. And, with human studies of gene therapies now underway for Leber congenital amaurosis (LCA, RPE65 mutations), Usher syndrome type 1B, Stargardt disease, retinoschisis, choroideremia and retinitis pigmentosa (MERTK mutations), scientists are gaining new knowledge every day about the best technologies and methodologies for gene-therapy development.
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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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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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Holiday Giving Means Doubling Your Gift

Allison Corona videoFor many reasons—not the least of which are holiday-related—December is a month when we all consider ways to give to others. And the spirit of giving has never been more apparent than it is right now at the Foundation Fighting Blindness. Not only is our end-of-year fundraising campaign in full swing; it’s bolstered by the Chairman’s Holiday Match. What that means is, for every dollar someone gives, it’ll be matched by another dollar—thus doubling its value. Give $25, it’s worth $50. Give $50, it’s worth $100. And so on.
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