Katie Myers' son, Kaden, has SMA or spinal muscular atrophy.
SMA, she says, can be a death sentence for babies.
"Dying from a rare disease is horrible and tragic. But dying from a rare disease when there should've been a treatment available-- it's unthinkable."
Katie says the drug, nusinersen, is currently used in clinical trials but not accessible to all children with SMA.
The group asked the FDA and companies behind the drug to make it quickly available to kids across the country.
"It's honestly sometimes harder than the diagnosis itself, knowing there's something out there that can help your kid but you can't do anything to access it," Katie said.
She said her 2-year-old son was never qualified to participate in trials due to his age, which is why she's been fighting to make the drug available to all children.
"There is a window with kids with SMA to get the most from that treatment and knowing that he's in that window now just makes it more important to us," she said.
Since their meeting last week, changes have already been made.
Before, the pharmaceutical companies marked success when a patient survived while on the drug.
Now, the FDA approved changes that now mark success when a child's motor skills improve.
"It's a long time to have to wait for a child to pass away from a disease to know if it's effective versus just seeing any increase in motor skills," Katie said.
She hopes this change will make the drug available on the market sooner than before, and urges other parents to fight for their children like she has.
"Your voice is important. Use it. Advocate," she said.
The FDA released this statement in response to the group's visit to D.C.:
The FDA recently had a productive and informative meeting with the SMA community in which we heard from families about the characteristics of SMA and learned about their concerns. The FDA shared information about our expedited approval pathways, discussed how to request expanded access to investigational drugs (compassionate use), and discussed the drug approval process in general. The SMA advocates explained their priorities and concerns, notably the willingness to accept more risk with investigational products and the challenges some patients have had with clinical trial enrollment.
We mentioned to the group that FDA regulations allow for flexibility in applying regulatory standards, particularly when considering applications for drugs to treat rare diseases. The FDA continues to use various expedited programs, such as breakthrough therapy designation, fast track, priority review, and accelerated approval to help promote innovation and to speed patient access to urgently needed safe and effective products. The FDA is fully engaged with drug companies that are developing new therapies for SMA. We hope that as we continue to work together there will be progress toward new treatment.