On Tuesday, December 20th, The U.S. Food and Drug Administration (FDA) approved LUXTURNA, what is hoped to be a one-time gene therapy product for patients with a confirmed biallelic RPE65 mutation-associated retinal dystrophy. We first mentioned LUXTURNA in our email on Thursday, October 26th. Please click here if you missed it! LUXTURNA is for patients with mutations on both copies of the RPE65 gene who also have sufficient viable retinal cells to undergo therapy. According to a press release by the manufacturer of LUXTURNA, Spark Therapeutics, it "is the first FDA-approved gene therapy for a genetic disease, the first and only pharmacologic treatment for an inherited retinal disease (IRD) and the first adeno-associated virus (AAV) vector gene therapy approved in the U.S." Speaking of the approval, VisionAmerica's Dr. Rod Nowakowski said, "FDA approval bears witness to two extraordinary events. First, the scientific process from hypothesis to results that culminated in approval and second, a marketable gene therapy for a visually devastating hereditary retinal dystrophy. Others are sure to follow, delivering on the promise of personalized (ophthalmic) medicine, with no end in sight. (Pun intended!)"
Genetic testing is the only way to determine whether or not the cause of a patient's IRD is related to the biallelic RPE65 mutations. As we mentioned last week, ID Your IRD, one of the only programs offering free genetic testing, is undergoing significant changes. If you missed our update, please click here to read last week's post. If you have questions related to this topic, please do not hesitate to contact us.
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AuthorThe staff and doctors at VisionAmerica are committed to providing relevant information for you, your patients and your practice. We hope you find the information in our blog post helpful. Archives
August 2019
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