Young patients seek for spinal muscular atrophy cure, to participate Lorain event Saturday

LORAIN, OH (WOIO) - On any given sunny day, 10-year-old Thomas Shepherd and his brother, 3-year-old George, head out to play in their cul de sac in Avon Lake.

Just like other kids, Thomas and George love going to Indians games and visiting Disney World. They don't let the fact that they are in wheelchairs hold them back.

Both brothers have what's called spinal muscular atrophy or SMA, a genetic disease that affects muscular movement.

One in 50 people are carriers for spinal muscular atrophy.

At 6-months-old, something just didn't seem right with Thomas. He wasn't meeting milestones typical for babies his age.

Thomas would eventually be diagnosed with SMA right after his first birthday.

"I just remember sitting in the parking lot crying," said Laura Shepherd, Thomas's mom.

Later, Thomas's little brother would be diagnosed with SMA too.

"A million things go through your head. All the different things they are not going to be able to do, and once you get over that point, you have to change your mindset to they can do everything anybody else can, just in a different way," added Shepherd.

There's new hope for patients like Thomas and George.

A new drug called Spinraza was approved by the FDA in 2016.

"I never thought that I would see this in my career - that a baby with Type 1 SMA would be sitting, standing, walking," said Dr. Nancy Bass, a pediatric neurologist for University Hospitals.

Doctors say, the sooner Spinraza is administered, the greater the outcome for patients.

Soon Infants will be tested for SMA when they are first born.

"It's been approved in multiple states, and it is very soon to be approved in Ohio, so then, you can catch a baby before they even get weak - start them on treatment and maybe, in a sense, get rid of these children that are losing those milestones," said Bass.

George and Thomas also have benefited from taking Spinraza, but it's not a cure.

More research is needed.

The Shepherds help to raise money for the organization Cure SMA every year. They will be walking in the Cure SMA Walk-n-Roll at Lakeview Park in Lorain this coming Saturday.

"To get these drugs for these rare diseases takes a lot of money because the fewer the patients, the harder it is to get a drug tested and approved so it's safe," said Matt Shepherd, Thomas and George's Dad.

In the meantime, the Shepherds live life to the fullest, hoping that someday there may be a cure.

Click here to register for this weekend's Cure SMA Walk-n-Roll or to donate to help find a cure and help families living with SMA.

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