Patients who had lost their sight to an inherited retinal disease could see well enough to navigate a maze after being treated with a new gene therapy, according to research presented today at AAO 2017, the 121st Annual Meeting ...
Nov 11, 2017
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(HealthDay)—A U.S. Food and Drug Administration advisory panel was poised on Thursday to recommend approval for a gene therapy that could grant the gift of sight to young people with a rare type of inherited vision loss.
Oct 12, 2017
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Approximately one in every 100 babies is born with congenital heart disease (CHD), and CHD remains the leading cause of mortality from birth defects. Although advancements in surgery and care have improved rates of survival ...
Oct 9, 2017
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The human population carries around more deadly genetic diseases than would be expected based on a simple comparison of mutation rates and deaths of affected individuals. Carlos Eduardo Guerra Amorim of Stony Brook University ...
Sep 28, 2017
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The first known identification of two genes responsible for hypoplastic left heart syndrome (HLHS), a severe congenital heart defect, has been reported by researchers at the University of Pittsburgh School of Medicine. The ...
May 22, 2017
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(HealthDay)—The U.S. Food and Drug Administration says it has expanded approval for the cystic fibrosis drug Kalydeco (ivacaftor) to include 33 mutations of the disease, up from the previous 10 mutations.
May 18, 2017
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Research published online in The FASEB Journal shows that the improper methylation of a protein called "Septin2," which regulates the structure of cilia, was associated with an increased risk of having a neural tube defect ...
May 11, 2017
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Researchers have identified a gene that plays a key role in the formation of neural tube defects, a problem commonly found in infants of pregnant women with diabetes. This is the first time the gene has been shown to play ...
May 5, 2017
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A group of researchers has found that three siblings born with cleft lip and palate share a common gene mutation associated with the birth defect.
Mar 21, 2017
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Using a new cellular model, innovative gene therapy approaches for the hereditary immunodeficiency Chronic Granulomatous Disease can be tested faster and cost-effectively in the lab for their efficacy. A team of researchers ...
Mar 21, 2017
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