"Since being on prednisone, I’ve been up and down with my weight and up and down with the milligram dosage,” says Carlie Brinker. “I’m 19, and I’ve been on prednisone for 11 years.”

In 1990, Sara Winokur was a doctoral student in the laboratory of John Wasmuth, a professor of biological chemistry and a prominent researcher in genetics at the University of California at Irvine.

It was an exciting time in genetics. The genes that, when mutated (flawed), cause diseases, were rapidly being identified. Among the first, in 1986, had been one for Duchenne muscular dystrophy.

It seems as if everybody wants information about you these days. Try to get that rebate the salesman promised on your new computer, and the company wants to know how old you are, how much money you make and whether you went to college.

Phone surveys want to know what you think of your congressional representative, whether you’re planning to sell your house and what radio stations you listen to.

The first U.S. human gene therapy trial directed at Duchenne muscular dystrophy (DMD) was launched yesterday at Columbus (Ohio) Children's Hospital, the Muscular Dystrophy Association (MDA), Children’s Hospital, and Asklepios Biopharmaceutical Inc. (AskBio) announced today. 

Neurologist Jerry Mendell administered an injection of AskBio’s Biostrophin, which contains a functional gene for the muscle protein dystrophin, into the biceps of Andrew Kilbarger, 8, of Lancaster, Ohio.

Just a few years ago, almost nothing could be said to parents like those in “Families Left with Questions,” other than that their child had a congenital (present at or near birth) form of muscular dystrophy.

Speakers at an MDA-sponsored workshop held June 14 in Tucson, Ariz., as part of a larger meeting of the Federation of American Societies for Experimental Biology (FASEB), presented a variety of reports about muscle stem cell experiments and clinical trials.

Myoblast transfer

Louis Kunkel, molecular geneticist and MDA grantee at Children's Hospital of Boston, revisited the myoblast transfer trials of the early 1990s.

In this article: research news about prednisone in Duchenne MD; tests for Duchenne and Becker MDs; and the latest progress in research for spinal muscular atrophy

In the late 1860s, 9-year-old Bridget Moore left Ireland for New York, bringing with her “a small steamer trunk, a pair of rosary beads, a clay pipe, and as near as we can figure out, Duchenne muscular dystrophy.” Now her great-granddaughter, Christine Kehl O’Hagan, has written a memoir about her family’s multigenerational history with DMD. The Book of Kehls, published by St. Martin’s this month, traces this bittersweet history with candor and humor.

When I was 18, I informed my doctor I'd be attending college in the fall.

Later, he asked to speak to my parents privately. He told them it was nice that I was going to college but, because I had Duchenne muscular dystrophy (DMD), they shouldn't expect me to survive to graduate.

Well, I finished college. I'm now almost 40 years old and working as a software engineer. Ironically, five years after making that statement, the doctor passed away.