Muscular dystrophy patients get first gene therapy
The Food and Drug Administration approved the first gene therapy for Duchenne muscular dystrophy, but limited access to those ages four and five.
muscular dystrophy
His baby gene editing shocked ethicists. Now he's in the lab again
He Jiankui, who shocked the world in 2018 by announcing the creation of the first gene-edited babies, tells NPR he's now working on a cure for Duchenne muscular dystrophy.
FDA advisers narrowly back first gene therapy for muscular dystrophy
A panel of experts voted 8-6 in favor of Food and Drug Administration approval of the first gene therapy for Duchenne muscular dystrophy, a fatal genetic disease.
Gene therapy for muscular dystrophy stirs hopes and controversy
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.
A Boy With Muscular Dystrophy Was Headed For A Wheelchair. Then Gene Therapy Arrived
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.
Duchenne Drug Delayed After Outrage Over Price
Many Duchenne muscular dystrophy patients in the U.S. have imported a medicine called deflazacort for about $1,200 a year. A brand-name version just approved for sale in America costs $89,000.
Are Golden Tickets That Speed Drugs Through FDA Worthwhile?
A voucher awarded to a company that finds a treatment for a rare childhood disease can be sold to the highest bidder and then used to fast-track the review of another drug. Are the prizes worth it?
Controversy Continues Over Muscular Dystrophy Drug, Despite FDA Approval
The Food and Drug Administration approved a muscular dystrophy drug despite deeply flawed evidence. Was the decision a dangerous precedent or flexible pragmatism reflecting patients' values?
Experimental Drug Jams Ebola Gene To Fight The Virus
A drug being tested against Ebola makes use of new scientific insights that could prove useful for treating other illnesses, including one that is inherited.