[Njtechdiv] History Is Made: FDA Approves Spark’s Vision-Restoring Gene Therapy
Mario Brusco
mrb620 at hotmail.com
Wed Dec 27 18:51:21 UTC 2017
if interested, I encourage anyone to read the full blog post at the
address below. also, consider to have yourself tested for the gene
mutation if you're interested in this matter with the advisement of your
ophthalmologist or vision specialist.
History Is Made: FDA Approves Spark’s Vision-Restoring Gene Therapy
http://www.blindness.org/blog/index.php/history-is-made-fda-approves-sparks-vision-restoring-gene-therapy/
By Ben Shaberman
on December 20, 2017
Spark Therapeutics’ vision-restoring RPE65 gene therapy has received
marketing approval from the U.S. Food and Drug Administration, becoming
the first gene therapy to gain regulatory approval in the U.S. for the
eye or any inherited condition.
Known as LUXTURNA™ (voretigene neparvovec), the gene therapy restored
vision in a clinical trial for people between the ages of 4 and 44 with
Leber congenital amaurosis (LCA) caused by mutations in the gene RPE65.
Study participants with severe vision loss reported putting away their
navigational canes, seeing stars, being able to read, and recognizing
faces of loved ones. Vision restoration has persisted for at least three
years. The treatment is also designed to work for people with retinitis
pigmentosa (RP) caused by RPE65 mutations.
...
“This is truly a historical moment in the fight against blindness. We
are delighted by the FDA’s approval of LUXTURNA for people with RPE65
mutations,” says Stephen Rose, PhD, chief research officer at FFB.
“LUXTURNA’s approval also provides affirmation that gene therapies can
be a safe, effective, and commercially viable approach to treating many
forms of blindness, providing a big boost to an already burgeoning
field. The approval is great news for a broad spectrum of people with
inherited retinal diseases.”
Retinal gene therapy clinical trials are underway for people with a wide
range of retinal diseases including: X-linked RP, choroideremia, Usher
syndrome type 1B, Stargardt disease, X-linked retinoschisis, and
achromatopsia.
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