Knoxville family celebrates ‘Rare Disease Day’ and new FDA-approved treatment

Local News

KNOXVILLE, Tenn. (WATE) — February 28 is “Rare Disease Day,” a day to raise awareness for people living with a rare disease and their families in hopes to improve access to medical treatments worldwide.

One of those rare diseases is called SMA.

SMA, or Spinal Muscular Atrophy, is a rare, progressive Neuro-Muscular Disorder that causes difficulty with basic life functions like breathing, swallowing, and walking.

Nora Gooden’s favorite colors are pink and black. The Knoxville native also has SMA.

“So Nora is 12,” said Nora’s mom, Jamie Gooden. “She just turned 12 in January. She is a 6th grader at Holston middle school, so this is our first year in middle school.”

Nora likes music and video games.

“She loves K-pop, and YouTube, and Road Blocks and anime,” adds Jamie.

Like most middle schoolers, you can find Nora surfing the web on most days.

“She’s pretty brave,” explained her mom. “She gets into a lot of stuff. She is a computer wiz.”

However, unlike some middle schoolers, Nora has a unique disorder.

“Nora was just a baby,” Jamie said. “So about 6 months old is when we were diagnosed with SMA.”

Nora and her parents have had to travel hundreds of miles for treatment.

They were traveling from Knoxville to North Carolina every four months for treatment, but thanks to a recently approved treatment, they can now stay home to administer her medications.

T.J. and Jamie Goodman explain:

T.J.: “That drive for us to Winston Salem its,”

JAMIE: “Wake Forest in Winston Salem.”

T.J.: “It was like, ten hours?”

JAMIE: “Well round trip about 12.”

T.J.: “We ended up buying a new van to make Nora more comfortable for that ride.”

But, with a recently FDA-approved treatment, the Gooden family doesn’t have to make the 12-hour trip anymore.

“August, we started the new drug that was released by the FDA called Evrysdi.,” said Jamie. “So now we can administer it at home.”

Thanks to this new treatment, Nora and her family can continue living a full life.

“One of the biggest takeaways from our story is that there is hope,” Jamie said.

Evrysdi is the first and only medicine for SMA that can be taken at home.

The Goodens said with Covid-19, being able to use the new treatment without having to travel and expose Nora to hospitals is a blessing.

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