She said with the success in Europe, there is now some hope the medicine might save her sons. But it’s been slow going.

Kulsrud said when she learned of the success in the European patients, she contacted a FDA again about getting her sons on it.

One of the patients began using the drug in May, she said.

“But the FDA is digging their heels in ... there is no movement at all,” she said.

Kulsrud also has met with North Dakota’s congressional delegation, but their efforts have yielded no results.

She was told by the drug manufacturer the FDA wants another study on the drug and possible side effects.

The company has already conducted one study at a cost of $3 million, she said.

PKAN typically strikes in childhood and is fatal in 10-12 years after being diagnosed. Martin Shkreli, co-inventor of RE-024, said only about 1,000 children in the U.S. and 6,000 worldwide have been diagnosed with the disease.

He said part of the problem is the FDA has no specific division to deal with what are known as “orphan diseases” — diseases that affect fewer that 200,000 patients.