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Posts tagged pharmaceutical

FFB Convenes Experts to Discuss Therapeutic Opportunities for Stargardt Disease

Meeting presenters (left to right): Philip Rosenfeld, MD, PhD; Ilyas Washington, PhD; Peter Charbel Issa, MD, PhD; Elias Traboulsi, MD, MEd; Ulrich Schraermeyer, PhD; Carel Hoyng, PhD; Paul Bernstein, MD, PhD; SriniVas Sadda, MD; Krzysztof Palczewski, PhD; Janet Sparrow, PhD; Artur Cideciyan, PhD; Hendrik Scholl, MD; Patricia Zilliox, PhD (not pictured)

Meeting presenters (left to right): Philip Rosenfeld, MD, PhD; Ilyas Washington, PhD; Peter Charbel Issa, MD, PhD; Elias Traboulsi, MD, MEd; Ulrich Schraermeyer, PhD; Carel Hoyng, PhD; Paul Bernstein, MD, PhD; SriniVas Sadda, MD; Krzysztof Palczewski, PhD; Janet Sparrow, PhD; Artur Cideciyan, PhD; Hendrik Scholl, MD; Patricia Zilliox, PhD (not pictured)

Stargardt disease is the world’s leading cause of inherited macular degeneration, affecting 30,000 people in the United States alone. It is also a challenging condition to understand and treat. While Stargardt disease often causes severe loss of central eyesight, its effect on vision and the retina can vary widely from patient to patient. The disease usually strikes in childhood or adolescence, but there are forms that cause significant vision loss much later in life. Also, a patient’s vision can remain stable for many years before a relatively sudden, steep decline occurs.

The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) convened 70 of the world’s top Stargardt disease research experts in Cleveland, Ohio, on February 17, 2017, to discuss these challenges and the current state of therapy development. Much of the information and data shared during the meeting came out of ProgSTAR, the FFB-CRI-funded natural history study of 365 Stargardt disease patients.
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ARVO 2016: Emerging Drug Targets Toxic Build-Up in Stargardt Disease

Dr. Hendrik Scholl at ARVO 2016.Like many diseases affecting the macula, the center of the retina, Stargardt disease is a waste-management problem. The “garbage” comes from the processing of vitamin A, which is an essential nutrient for vision. If you think of the retina as the engine for vision, vitamin A is the fuel; it enables the retina to convert light into the electrical signals that enable vision. And just like gasoline, which produces exhaust, vitamin A, when metabolized, leads to undesirable by-products.
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