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Gene Therapy for Retinal Dystrophy Approved by the FDA

posted by Fnord666 on Wednesday December 20 2017, @06:42AM   Printer-friendly
from the jean-therapy dept.

takyon writes:

The Food and Drug Administration (FDA) has approved a gene therapy to treat RPE65-mutation associated retinal dystrophy. But it will be expensive:

A first-of-its-kind gene therapy received approval from the Food and Drug Administration on Tuesday to treat a rare, inherited form of childhood blindness.

The FDA marketing clearance of Spark Therapeutics's Luxturna is historic for scientific and financial reasons. Luxturna is the first gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.

A Spark spokesman said the company will not disclose the Luxturna price tag until early January. Wall Street analysts expect the gene therapy to command a $1 million price tag—another first, but not necessarily a welcome one. At a time when drug prices are coming under intense scrutiny, Spark will need to convince insurers, politicians, and pharma critics that the benefit to patients offered by Luxturna justifies its high cost.

Also at NPR.

Previously: FDA Approves a Gene Therapy for the First Time
FDA Committee Endorses Gene Therapy for a Form of Childhood Blindness

Original Submission

 
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  • (Score: 3, Insightful) by Crash on Wednesday December 20 2017, @07:12AM (2 children)

    by Crash (1335) on Wednesday December 20 2017, @07:12AM (#612190)

    $250,000 Insurance Deductible...

    Choose One:
    1) Stay Blind.
    2) Be Homeless.
    3) Marry into the Koch Family.

    How does that go?

    "Give me your tired, your poor, Your huddled masses yearning to breathe free, The wretched refuse of your teeming shore. Send these, the homeless, tempest-tossed to me. I lift my lamp beside the golden door."

    At least we can still breathe free.

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  • (Score: 2) by c0lo on Wednesday December 20 2017, @07:32AM

    by c0lo (156) Subscriber Badge on Wednesday December 20 2017, @07:32AM (#612197) Journal

    I lift my lamp beside the golden door

    And... Retinal distrophy sufferers can't see it, as it can't be seen even by normal eyes: the golden door was plundered long ago, you'll find the pieces in Bahamas and the like accounts.

    --
    https://www.youtube.com/@ProfSteveKeen https://soylentnews.org/~MichaelDavidCrawford

  • (Score: 2) by DeathMonkey on Wednesday December 20 2017, @06:37PM

    by DeathMonkey (1380) on Wednesday December 20 2017, @06:37PM (#612439) Journal

    At least we can still breathe free.

    Nah, they're fucking with the EPA too...