By Roslyn Anderson, June 4, 2025 | From: WLBT

RANKIN COUNTY, Miss. (WLBT) - The Food and Drug Administration is holding a drug in the balance that one family says improves the lives of those with rare diseases.

One Rankin County family says the medication Elamipretide has given one teenager the strength to be a normal kid, and they don’t want that taken away.

“It’s helped a lot. Before, I couldn’t go anywhere and keep up with my friends,” said Christopher Pena.

The 17-year-old suffers from Barth Syndrome, a rare metabolic disease that impacts the muscles and immune system. He takes Elamipretide. The Food and Drug Administration has delayed approval after years of trials.

Video shows Christopher slowly struggling to climb stairs before the drug, and this month’s video shows him easily walking up stairs during a recent ceremony at Brandon High School.

“The FDA is making it where there’s a chance for us not to be able to take it,” said Christopher. “It’s gonna make it where a lot of things I’m able to do now, can’t like I can’t do them anymore.”

When Christopher was born, his mother, Kristi Pena, said doctors said there was no hope for him. He was in hospice care before his first birthday.

In November, he began Elamipretide, a treatment she said changed everything. Now, the vigilant mother is pleading with the FDA to work with Stealth Bio Therapeutics to approve the drug. She said the medication enables her son to now live an active life.

“Not long after starting that drug, he noticed that he was able to jump with both feet leaving the ground, which is another thing he’s never been able to do, and I can’t credit that to any other reason but this medicine,” said Kristi Pena.

Her sister is Amy Wilson. Wilson’s son also has Barth Syndrome and was in the clinical trial for Elamipretide until he developed a minor rash.

“He is not on it now. His health has declined. He now has a feeding tube,” said Wilson. “He’s lost significant amounts of weight. His kidney function has declined to the point where he didn’t even feel well enough to get out of the bed and come to this interview.”

The family will know in about six months if the FDA approves the company’s application to continue producing the drug.

They also want to meet with Senator Cindy Hyde-Smith to encourage the new FDA Commissioner Marty Makary to urge the agency to focus on approving medical treatments for patients with ultra-rare diseases.