Single Use Gene Therapy Approved in the EU for Leber’s Congenital Amaurosis
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Single Use Gene Therapy Approved in the EU for Leber’s Congenital Amaurosis

According to a story from pm360online.com, the drug developer Novartis has recently announced that the company's gene therapy Luxturna has gained approval in the EU as a treatment for Leber's…

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ICYMI: FDA In Favor Of Luxturna, A Gene Therapy For Leber Congenital Amaurosis
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ICYMI: FDA In Favor Of Luxturna, A Gene Therapy For Leber Congenital Amaurosis

The FDA, US Food and Drug Administration, unanimously agreed upon the recommendation for a new gene therapy that treats a rare form of blindness, reported ScienceNordic. Spark Therapeutics, a biotech…

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Gene Therapy Luxturna Will Cost $850,000 To Patients With Rare Eye Disease
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Gene Therapy Luxturna Will Cost $850,000 To Patients With Rare Eye Disease

The FDA passed along their nod of approval for the sought after gene therapy treatment, Luxturna, reported Engadget. This will be used to help aid those made blind by a…

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ICYMI: FDA Makes History Aproving a Gene Therapy That Can Improve Childhood Blindness
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ICYMI: FDA Makes History Aproving a Gene Therapy That Can Improve Childhood Blindness

According to The Washington Post, on Tuesday the Food and Drug Administration, FDA, made history by approving a gene therapy for childhood blindness, which will change the lives of many…

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Patients of Inherited Diseases Just Got a Christmas Miracle!
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Patients of Inherited Diseases Just Got a Christmas Miracle!

Gene therapy has been on the horizon for years now, but before today, it was only a reality for patients with non-inherited diseases caused by genetic mutations. That just changed!…

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Luxturna Gene Therapy Could Change the Game in Treating Inherited Blindness
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Luxturna Gene Therapy Could Change the Game in Treating Inherited Blindness

On this season of America's Got Talent, the world met Christian Guardino. I was one of millions who heard his beautiful singing voice as he belted out his song onstage.…

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Company Developing Treatment for Rare Childhood Blindness Gets Special FDA Designation
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Company Developing Treatment for Rare Childhood Blindness Gets Special FDA Designation

The FDA has granted a special designation for a drug company developing a gene therapy for the treatment of leber congenital amaurosis (LCA). MeiraGTx received rare pediatric disease designation for its…

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Will Single Injection Offer Improvements for Leber Congenital Amaurosis?
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Will Single Injection Offer Improvements for Leber Congenital Amaurosis?

When something in our computers doesn’t work or has a defect, we download a “patch” from the manufacturer, and call it repaired. With ongoing scientific research and development, the practice…

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PBS Documentary “The Gene Doctors” Brings Rare Treatments to the Public Eye
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PBS Documentary “The Gene Doctors” Brings Rare Treatments to the Public Eye

Earlier this month, PBS released the "The Gene Doctors." The hour-long documentary follows the recent emergence of gene therapy as a novel method of treating rare diseases. The film discusses…

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“Fishing” for a Cure for Leber Congenital Amaurosis
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“Fishing” for a Cure for Leber Congenital Amaurosis

By examining a zebrafish, scientists have found the gene that affects the maintenance of cone photoreceptors and visual functions in the eye. It might be the knowledge that’s been needed…

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A New Treatment May Be Coming for Leber Congenital Amaurosis
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A New Treatment May Be Coming for Leber Congenital Amaurosis

Case Western Reserve University researchers have recently developed gene-carrying nanoparticles that are able to bring healthy genes to those negatively affected by Leber congenital amaurosis (LCA), a disease that causes…

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