The Latest Breaking News from Gene Therapy

Sarepta won approval Thursday for its highly anticipated Duchenne muscular dystrophy gene therapy. SRPT stock yo-yoed in response.

Uniqure unveiled mixed results for its study of a Huntington's disease treatment on Wednesday, and QURE stock crashed to a six-year low.

The FDA pushed back its review of a highly anticipated gene therapy from Sarepta Therapeutics on Wednesday, prompting SRPT stock to tumble.

The U.S. Food and Drug Administration approved a gene therapy for dystrophic epidermolysis bullosa, also known as butterfly disease.

Gene therapy has helped doctors make major strides in treating inherited blindness, and it's now being explored for other conditions.

Shares of SRPT stock launched Monday after FDA advisors voted late Friday in support of a highly anticipated gene therapy from Sarepta.

An estimated 295 million people suffer from visual impairment globally. Around 43 million of those people are living with blindness. While not every form of blindness can be cured, recent scientific breakthroughs have uncovered new ways to treat some forms of inherited blindness through gene therapy.

Nicola Kelleher's partner Bradley Benjamin has a hereditary kidney disease which has seen him suffer on dialysis for years - and they didn't want the same outcome for their child

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.