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The teen was born with a rare genetic condition that causes blisters all over his body and in his eyes. But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. His doctor wondered if it could be adapted for his eyes.
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The Food and Drug Administration approved the first gene therapy for Duchenne muscular dystrophy, but limited access to those ages four and five.
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The FDA is considering greenlighting the experimental treatment under its accelerated approvals program. Some critics point out the therapy isn't yet proven to work and may be costly.
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The first brain-delivered gene therapy on the market was recently approved in Europe and the U.K. for a rare genetic disorder called AADC deficiency. It causes developmental delays and movement disorders in kids.
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Although it’s still unclear what killed the 27-year-old, his death is raising questions about the experiment and the overall prospect of what one ethicist calls designer genetic therapies.
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Gene therapy has helped a 9-year-old boy regain enough muscle strength to run. If successful in others, the treatment could change the lives of thousands of children with Duchenne muscular dystrophy.
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The Food and Drug Administration approved a new gene therapy for a rare but devastating genetic disorder. The drugmaker says the cost is worth it because it's a one-time treatment that saves lives.
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Installment plans and refunds for treatments that don't work are two options getting more attention as ultra-expensive therapies become more common. The financial strains will only grow.
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U.S. health officials are eliminating special regulations for gene therapy experiments, saying that what was once exotic science is quickly becoming an…
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A first-of-its kind genetic treatment for blindness will cost $850,000 per patient, making it one of the most expensive medicines in the world and raising…