Living and Dying With Muscular Dystrophy


A Dutch biotech firm, Prosensa, is conducting human clinical trials of a therapy called exon skipping. This approach would transform DMD into the much milder form of Becker muscular dystrophy, which may allow sufferers to continue walking into their 40s.

Another biotech company, in North Carolina, Asklepios, is conducting a replacement therapy trial that aims to get a healthy copy of the defective gene into all the body's muscle cells, thus preventing the muscular and mortal effects of the disease. These research scientists say they are on the cusp of a cure.

Toward the end of "Darius Goes West," another young man is introduced. His name is Charley Seckler. He's about 6 years old and also has DMD. His father, Benjy, is a doctor. His mother, Tracey, is a teacher. Charley is still kicking a soccer ball, albeit slowly. They've started Charley's Fund to financially assist scientists around the world in their pursuit of a cure for DMD.

They told me that their own son might yet benefit from a breakthrough. After attending the premiere, they wondered whether he, too, would be able to stand in the ocean as a teenager, without being held up by his parents.

Of course, all these magnificent scientific breakthroughs will come too late for Darius -- and Tommy. But in their own ways, they have defied the odds, confronted insurmountable challenges and displayed courage beyond compare.

As I left the premiere, I suddenly remembered that I had watched a documentary on my last visit to Santa Barbara, back in 2005. I had been subpoenaed to appear in court during the Michael Jackson child abuse proceedings of 2005. As I sat in the dock, my documentary "Living with Michael Jackson" was played before the jury. I remain proud of that documentary, and I recall seeing even Judge Melville subtly nodding his head to some of Jackson's magnificent music.

But when I watched "Darius Goes West" something altogether more profound happened: I saw my brother again. And for that alone, I shall always be grateful. Click here to read about the latest developments in Darius Weems' life.

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