Story includes research items about: centronuclear myopathy; Duchenne, limb-girdle, and Emery-Dreifuss muscular dystrophies; and spinal muscular atrophy.

In the era of molecular biology, the drug development process has moved from a “let’s try it and see what happens” approach to a scientifically based process of discovery and application.

For many of the diseases in MDA’s program, drug discovery begins with gene discovery — identifying a gene that, when flawed, causes a disease.

It was 5 o’clock on a weekday afternoon. I was sitting on the corner of Montrose, a five-lane thoroughfare through downtown Houston, and Sul Ross, a little cross street between West Alabama and Richmond. The class I was taking at the University of St. Thomas had ended for the day, and I was waiting for my faithfully late METRO bus.

*Many people interviewed for this article asked not to be identified to protect the privacy of affected family members.

“Everybody knows the word apathy,” says a California woman whose 25-year-old daughter’s type 1 myotonic dystrophy (MMD1, sometimes called DM1) was diagnosed just a few years ago. “People use the word loosely. I don’t think it does justice to the reality of this disease.”

A European group of researchers successfully used a combination of genetic correction and stem cells to treat Duchenne muscular dystrophy (DMD) in mice with the disease. The compound they used for the genetic correction was developed by Judith van Deutekom, then at Leiden (Netherlands) University Medical Center, with MDA support.

DMD results from the loss of the muscle protein dystrophin because of any of a number of genetic errors in the dystrophin gene.

In the early 1990s, amid great excitement, six trials took place of transplants of immature muscle cells from healthy relatives into boys with Duchenne muscular dystrophy. (Five were funded by MDA.)

On two different occasions, when Aaron came home from MDA summer camp and asked what was new, I’ve blurted out the news that we were moving. Now, years later, it’s a joke, but back then Aaron swore he would never go to camp again.

What a magnificent tribute to the gutsiness of eight incredible athletes!

On Oct. 14, after days of intense competition, Team USA emerged victorious in the Power Soccer World Cup in Tokyo.

On their way to the top they beat world-class power soccer teams from Belgium, Denmark, England, France, Japan and Portugal. Their final win came in a sudden-death overtime match against France, which had never before been beaten in international competition.

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 working at a stressful job an hour away from her home in Stony Point, N.Y. To her knowledge, there is no history of muscle disease in her family.