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Gonvick boy fights for life-saving drug

FARGO -- "I feel very angry and sad. I can't even find the words to say how mad I am," said Jacob Gunvalson. The 16-year-old from Gonvick, Minn., has Duchenne muscular dystrophy, and his anger stems from what he said was a promise made and broken...

FARGO -- "I feel very angry and sad. I can't even find the words to say how mad I am," said Jacob Gunvalson.

The 16-year-old from Gonvick, Minn., has Duchenne muscular dystrophy, and his anger stems from what he said was a promise made and broken by PTC Therapeutics, a New Jersey drug company.

PTC Therapeutics is testing a drug called PTC124, which Gunvalson and his parents believe shows promise of extending the lives of people with DMD.

Typically, those who have the disease die in their late teens or early 20s.

On Wednesday, Gunvalson and his mother, Cheri, told their story to a radio audience, broadcasting from West Acres shopping center on Al Aamodt's Mid Day show on WDAY AM.

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Cheri Gunvalson said she began pushing for muscular dystrophy research funding after her son was diagnosed in 1999.

With help from U.S. Rep. Collin Peterson and the late Sen. Paul Wellstone, both Democrats from Minnesota, Gunvalson said muscular dystrophy centers were established around the country that now carry Wellstone's name.

Gunvalson said PTC Therapeutics, which she said has received millions in federal research dollars, promised her son would have access to the drug.

She said a company executive advised her against having her son take part in a short-term trial and assured her that would not exclude him from long-term testing.

But 26 boys who participated in a 28-day trial have been moved into a 96-week trial her son was not allowed to join, she said.

Gunvalson said the decision bars her son from the drug until results are evaluated, which will take years.

"When you get the drug so late, it doesn't do any good," she said.

The family is suing PTC Therapeutics with the help of Mike Hatch, former Minnesota attorney general.

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Hatch, who took the case pro bono, said he will ask for an injunction that would force the company to provide Jacob Gunvalson with the drug until the suit is decided.

He said in some situations federal rules allow access to drugs before they are approved, but the company said it won't allow it in this case because it would have to give it to others who have asked for it.

Hatch said he believes PTC Therapeutics doesn't want to deal with the red tape that comes with granting an exception.

A statement released by PTC Therapeutics said the company never promised the Gunvalsons access to the drug.

"With only 28 days of safety data in DMD patients, we firmly believe it is too early to make the drug available on an individual basis outside of a clinic trial," the statement said.

Pat Furlong, president of the Parent Project Muscular Dystrophy, said parental desire for a cure can trump safety concerns, but she said the lawsuit could jeopardize the drug's approval for widespread use.

"I actually feel very saddened by this lawsuit," said Furlong, who lost two sons to DMD, one at age 15 and one at 17.

Furlong said she worries that if someone suffers an adverse effect from the drug, the approval process "would come to a grinding halt."

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Cheri Gunvalson said she knows what will happen if her son doesn't get PTC124.

"To have hope and then have the hope pulled out. There are no other options. There are no other drugs in the pipeline," she said.

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The Forum of Fargo-Moorhead and the Herald are Forum Communications Co. newspapers.

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