gene therapy

On this episode, an infant with a harrowing diagnosis defies all odds after receiving a $2 million genetic treatment. Then, a look at medical certifications for pilots.

About 350 million people suffer from rare diseases, most of which are genetic. But the individual disorders affect relatively few people, so there’s little commercial incentive to bring these treatments to market.

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.

The Food and Drug Administration approved the first gene therapy for Duchenne muscular dystrophy, but limited access to those ages four and five.

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.

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.

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.

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.

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.

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.