Skip to content
My WebMD Sign In, Sign Up

Health & Parenting

Font Size

Gene Therapy May Help Muscular Dystrophy Patients

Research May Lead to Future Treatments for Duchenne Muscular Dystrophy

First Human Tests continued...

He says there are other approaches to treatment in development. "Among the different things that are out there, for me, this is the most straightforward, the least plagued with toxic reactions, and the most immediately promising."

None of the patients appeared to see any clinically significant benefits from the study medication. But study researcher Franceso Muntoni, a pediatric neurologist and professor of medicine in the Institute of Child Health at University College London, says some patients noticed that they had better stamina.

"However, it is important to be aware of the placebo effect in short studies like this," he says.

And he says that because of how the protein works, patients wouldn't necessarily see immediate benefits.

"Dystrophin is a protein that does not make muscle stronger. It protects muscles from further breakdown, as would a shock absorber in our cars," Muntoni tells WebMD.

There were three serious adverse events reported in the study. Two, a case of vomiting after anesthesia and a broken ankle, were judged to be unrelated to the treatment. The third, a serious heart problem, was possibly related to treatment, but may also have developed as part of the underlying disease.

Muntoni says they are already working on putting together a larger study that would follow patients for a longer period of time.

He says it's the first time they've been able to try to correct the specific, underlying genetic defect in human patients. "This is remarkable and, until a few years ago, completely unforeseen."

1|2

Today on WebMD

Girl holding up card with BMI written
Is your child at a healthy weight?
toddler climbing
What happens in your child’s second year.
 
father and son with laundry basket
Get your kids to help around the house.
boy frowning at brocolli
Tips for dealing with mealtime mayhem
 
mother and daughter talking
Tool
child brushing his teeth
Slideshow
 
Sipping hot tea
Slideshow
Young woman holding lip at dentists office
Video
 
6-Week Challenges
Want to know more?
Chill Out and Charge Up Challenge – How to help your tribe de-stress and energize.
Spark Change Challenge - Ready for a healthy change? Get some major motivation.
I have read and agreed to WebMD's Privacy Policy.
Enter cell phone number
- -
Entering your cell phone number and pressing submit indicates you agree to receive text messages from WebMD related to this challenge. WebMD is utilizing a 3rd party vendor, CellTrust, to provide the messages. You can opt out at any time.
Standard text rates apply
Which Vaccines Do Adults Need
Article
rl with friends
fitSlideshow
 
tissue box
Quiz
Child with adhd
Slideshow