The U.S. Food & Drug administration recently approved a new drug for people with spinal muscular atrophy (SMA). The debilitating disease can be deadly, but a medicine called Spinraza could stop it from progressing.
The medication is something people with spinal muscular atrophy have waited years for, and a Tipton family says this is their first sign of hope as their son is set to get his first dose at Riley Hospital for Children at IU Health.
"We were told that he would probably never walk and that there were no treatments and no cure," said Anastasia Davis, whose son, Alexander, was diagnosed with SMA as a 1-year-old.
The rare genetic disease causes weakness and loss of motor skills as it progresses over time. Trials showed Spinraza stopped the advancement of the disease and even improved movement in some patients. The FDA approved the drug in December.
"I feel very scared and excited at the same time. I'm very nervous that it's not going to work," said Alexander, who's now 9 years old.
His mom and dad believe this drug will change their lives.
"Any little improvement with strength will help. Anywhere from being able to hold his head up better to being able to swallow being able to cough when he's sick," said Sean Davis, Alexander's father.
An optimistic outlook the Davis family wants to share with other families dealing with SMA.
"That there's hope. Even when it seems like the worse thing in the world these kids are amazing kids."
SMA is the most common genetic cause of death in infants. This is the first approved treatment for the debilitating disease.
To follow Alexanders journey check out his Facebook page Hakuna Matata for Alexander.