gene therapy

This is the story of a fatal genetic disease, a tenacious scientist and a family that never lost hope.

Conner Curran was 4 years old when he was diagnosed with Duchenne muscular dystrophy, a genetic disease that causes muscles to waste away.

Conner's mother, Jessica Curran, remembers some advice she got from the doctor who made that 2015 diagnosis: "Take your son home, love him, take him on trips while he's walking, give him a good life and enjoy him because there are really not many options right now."

The federal Food and Drug Administration has approved a gene therapy for a rare childhood disorder that is now the most expensive drug on the market. It costs $2.125 million per patient.

But for those patients lucky enough to get it, it appears it can save their lives with a one-time treatment.

Three-year-old Donovan Weisgarber is one of those patients. When he was born he seemed perfectly healthy. But within weeks, it became clear something was terribly wrong.

Researchers expect that three dozen new drugs will come on the market over the next few years with astronomical prices — some likely topping a million dollars per patient.

The drugmaker Novartis has told investors it might be able to charge $4 million to $5 million for one of its potential products, a treatment for a rare disease called spinal muscular atrophy.

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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 established form of medical care with no extraordinary risks.

Daylina Miller/Health News Florida

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 questions about the affordability of a coming wave of similar gene-targeting therapies.

The Food and Drug Administration Tuesday approved the first gene therapy to treat an inherited disease.

The treatment is called Luxturna, a genetically modified virus that ferries a healthy gene into the eyes of patients born with retinal dystrophy, a rare condition that destroys cells in the retina needed for healthy vision.

Eli Wheatley and Christian Guardino are among a growing number of patients whose lives are apparently being saved or radically improved by gene therapy.

Wheatley, 3, of Lebanon, Ky., and Guardino, 17, of Patchogue, N.Y., were both diagnosed with what were long thought to be incurable genetic disorders. In the past, Wheatley's condition would have probably killed him before his first birthday. Guardino's would have blinded him early in life.

But after receiving experimental gene therapies, both seem to be doing fine.

UF Health

Researchers at the University of Florida have developed a new therapy that they hope could lead to a cure for multiple sclerosis.

The Food and Drug Administration on Wednesday announced what the agency calls a "historic action" — the first approval of a cell-based gene therapy in the United States.

The FDA approved Kymriah, which scientists refer to as a "living drug" because it involves using genetically modified immune cells from patients to attack their cancer.