A mother is preparing to make her case to federal officials in Washington, D.C. to approve a drug that might help her children.
On Monday, Jen Peters is expected to speak before the U.S. Food and Drug Administration and share what it is like to have two children living with an ailment that robs them of their ability to use all of their muscles.
Grayson, 3, lacks the ability to clear his own mouth because he cannot swallow, Peters says.
"We have to suction him quite frequently," says the mother of three. “He has to lay flat to be able to manage his secretions. He can't hold his head up. He can't swallow, he can't eat anything.”
Before he was born, doctors diagnosed him with Spinal Muscular Atrophy – also known as SMA – meaning his nerves cannot communicate with his muscles because he is missing a protein that helps the brain tell his body to move.
His sister Ella, 6, also has it.
“She can use her arms. She just can't use her legs. She can hold her head up and eat,” Peters says.
Ella has SMA Type 2 while her brother is Type 1, however both need a machine to help them breathe while they are sleeping.
Fed up with the fact there is no treatment available in the market, the mother joined other families last fall to start a movement.
“Our goal in organizing FAST has been to educate our community on where the drug trials are,” Peters says. “How can it affect us – what can we do with it.”
The FAST movement, Families for the Acceleration of SMA Treatments, plans to urge the FDA officials to approve a treatment called Nusinersen, made by pharmaceutical company Ionis.
It is currently undergoing clinical trials in infants and young children
Peters says she has seen stellar results with some of the treatment patients and believes now is the time for it to become accessible to other SMA families.
“There's always that fear that right now – is it what I'm going to have in a year? Ella might not be able to hold her up anymore. Or Grayson might not be able to smile anymore,” Peters says. “Many families are faced with that very thing right there.”