Spinal Muscular Atrophy, SMA, is a rare condition that causes newborns to lose their ability to move their arms, legs, fingers and toes.

Without early treatment, most babies will not live to see their second birthday.

But now, there's new hope. An oral medication that can be given at home that literally stops this disease in its tracks.

When little Ryu was born in the Philippines, everything seemed perfect. Ryu's mother, Hazel, said at eight weeks old, he was losing muscle strength in his legs.

With limited access to genetic testing, it took months to figure out what was happening.

Diagnosed with SMA, parents John and Hazel decided to come to the United States, where new treatments could help ave baby Ryu.

"Because the nerves aren't speaking to the muscle, then the muscle becomes weak," Child Neurologist Ann Tilton said.

SMA is caused by a defect in the survival motor neuron one-gene.

"These genes allow the motor neuron to survive. So, if you don't have that, then you lose the nerves. The most efficient one is called SMN 1, that's what's missing in Spinal Muscular Atrophy," Tilton said.

Now, the first FDA approved oral medication to treat SMA, Evrysdi, can turn on the second SMN gene, making it more active.

Tilton can hardly believe what she's seeing.

"It's absolutely remarkable that they have developed a medication that can turn on a specific gene to alter the natural course of a disease. I mean, what a world, it's just absolutely miraculous," Tilton said.

Another medication was approved for SMA in 2016, but it involves a spinal tap every four months.

Evrysdi is the first and only oral medication approved by the FDA.

SMA is a genetic disorder that requires both mother and father to carry the gene. About one in 50 people in the U.S. actually have the potential of carrying it, which is why now, nearly all U.S. states are screening newborns for SMA.

Given the progressive nature of the disease, early awareness and treatment are critical.