Category Archives: retinitis pigmentosa

30Apr / 2015

AGTC Tackles 3 Eye Diseases with Gene Therapy

September will be 25 years since the first gene therapy experiment, and FDA approval is finally in sight. Several gene therapies are approaching the finish line, awaiting results from comparisons to existing therapies and analyses of long-term efficacy. Among the … Continue reading »

The post AGTC Tackles 3 Eye Diseases with Gene Therapy appeared first on PLOS Blogs Network.

Posted by in achromatopsia, ADA deficiency, adrenoleukodystrophy, Applied Genetic Technologies Corp, Foundation Fighting Blindness, gene therapy, giant axonal neuropathy, hemophilia, LCA2, retinitis pigmentosa, retinoschisis, Sanfilippo syndrome A, SCID-X1

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19Jun / 2014

Catching Up With 3 Rare Disease Families

 

Four-year-old Eliza O’Neill’s viral videos, the subject of my last two blog posts, continue to dominate the news media with another appearance on The Today Show June 17. Hopefully, her family’s fight to fund gene therapy for her …

The post Catching Up With 3 Rare Disease Families appeared first on PLOS Blogs Network.

Posted by in Batten disease, dna, fundraising, gene therapy, giant axonal neuropathy, Leber congenital amaurosis, NORD, rare diseases, retinitis pigmentosa, Sanfilippo syndrome

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