Meet Dr. Steve Rose
Steve is highly respected for his expertise and tireless commitment to finding treatments and cures for vision-robbing retinal diseases.
Archive for the ‘Research’ Category
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.
Definitive diagnoses for inherited retinal diseases don’t always come easy, even for the patients of the most knowledgeable doctors. Comments posted to this blog over the past year are a testament to that fact. Many readers are understandably frustrated by a doctor’s inability to determine exactly what retinal condition is affecting them or loved ones.
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Found in Translation: Advancing Treatments Into Human Studies
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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Two 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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Piggybacking on last week’s post by our CEO, Bill Schmidt, I wanted to tie two personal stories to exceptional advances made in retinal research in 2012. I’d be remiss if I didn’t mention that these advances were made possible by the generous support of FFB donors, and that future advances – which, eventually, will help 10 million Americans alone – depend on our donors’ continued support. I’ll revisit fundraising later in this post, but, first, you’ll find the first of the two stories – on how gene therapy restored some of a boy’s sight – below. The next story will be posted later this week.
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