Family sues co. for muscular dystrophy drug

The boy's mother, Cheri Gunvalson, who helped persuade Congress to significantly boost spending to find a cure for the disease, filed a lawsuit along with her husband and son in federal court in New Jersey this week.

Jacob Gunvalson, 16, suffers from Duchenne muscular dystrophy, a genetic, degenerative disease that mostly affects young boys. Typically, those who suffer from it die in their 20s because of weakness in their heart and lung muscles. There is no known cure, but the Gunvalson family believes the experimental drug holds hope.

The family claims that PTC Therapeutics of South Plainfield, N.J., repeatedly promised them Jacob would have access to the drug, known as PTC124, but then broke its word. The company denied ever making that assurance.

Jacob, who lost his ability to walk about a year ago, said in an interview that he thought he was going to get the drug many times.

"To think you can get help, and then just in the last second it's pulled out--that's like getting your whole future hopes and all that smashed," he said Thursday.

Jacob hopes to one day become a psychiatrist or psychologist to help people facing "tough times."

"But it takes a lot of schooling to get done with that," he said, "and I don't know how much longer I have."

Chip Baird, PTC Therapeutics' chief financial officer, said the company sympathizes with the Gunvalsons and other families suffering from the disease, but that PTC has always communicated with the Gunvalsons "in a clear and consistent manner."

In 2001, Cheri Gunvalson teamed with the late Sen. Paul Wellstone, D-Minn., for passage of the MD CARE Act, which increased federal funding to study the disease. The legislation established several "centers of excellence"--since renamed "Wellstone Centers"--for muscular dystrophy research.