Eric Hoffman

Compounds designed to alter genetic instructions for dystrophin, increase production of utrophin, or prevent scar tissue formation in muscle are moving through the development pipeline

posted on November 5, 2014 - 4:03pm
Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014. PTC is moving forward with ataluren
posted on March 31, 2011 - 11:41am
QUEST Vol. 18, No. 2
"I was always interested in the application of genetics to human problems," says Eric Hoffman, a molecular geneticist and MDA research grantee who directs the Center for Genetic Medicine Research at Children's National Medical Center in Washington. In the mid-1980s, as he was finishing his doctorate in biology at Johns Hopkins University, Hoffman says, "I began looking around for a human...

Blocking a protein associated with inflammation and scarring helped mice with a DMD-like disease

posted on May 22, 2009 - 4:23pm
A protein called osteopontin has been implicated as a cause of some of the detrimental inflammation and scarring ("fibrosis") of muscle tissue that takes place in Duchenne muscular dystrophy (DMD). Eliminating osteopontin was beneficial to mice with a DMD-like disease, and the researchers concluded that reducing osteopontin should be investigated as a possible therapy for DMD.

Success of 'exon-skipping' strategy in dogs bodes well for treatment of human DMD

posted on March 17, 2009 - 9:00pm
Scientists at Children's National Medical Center in Washington, Carolinas Medical Center in Charlotte, N.C., and the National Center of Neurology and Psychiatry in Tokyo, have successfully treated dogs with a disease closely resembling Duchenne muscular dystrophy (DMD) , using a molecular treatment strategy called "exon skipping." The strategy is simultaneously under development in human patients.
posted on May 1, 2008 - 9:39am
QUEST Vol. 15, No. 3
MDA has an Ace in celebrity supporter Singer, Grammy-nominated songwriter and actor Ace Young has a notable repertoire of philanthropy for his 27 years. Now he’s added MDA to the list of charitable endeavors he wants to help.

Duchenne muscular dystrophy carriers carry on, despite uncertainty

posted on November 1, 2007 - 4:20pm
QUEST Vol. 14, No. 6
When Rena Szymanski turned 40, she expected to slow down a bit. She never had been athletic, and it sometimes seemed to her that climbing stairs was harder for her than it was for other people, but she thought her strength was “normal” in general. Soon after her birthday, though, she noticed increasing weakness. “I didn’t know what was wrong with me,” says Szymanski, who until recently was...