There’s a disease where patients are missing the nerve cells in the spine that tells muscles to move.
A recently approved drug and a new delivery system are giving people hope.
The FDA recently approved the first-ever drug for spinal muscular atrophy, a degenerative muscle disease.
Shawn Stewart was diagnosed with spinal muscular atrophy when he was three.
Shawn Stewart says, ” I was walking until the age of 13. I broke my leg at that age. After that, I was no longer able to walk.”
He lost function in his arms, legs, and back. Just breathing became difficult.
For more than 45 years, doctors only treated his symptoms.
Then in 2016, the FDA approved Spinraza, a drug that increases the production of the SMN protein needed for muscle control.
Michael Muhonen, Neurosurgeon at St. Joseph Hospital says, “I’ve had patients that had no arm function that can now move their arms. I’ve had patients that were ventilated, couldn’t breathe on their own, and they can now breathe.”
The problem was that many SMA patients like Shawn have had a spinal fusion.
Giving the drug through a spinal tap three times a year was painful and potentially dangerous. So doctor Muhonen came up with this.
Muhonen continues, ” This is a port that allows us to inject the Spinraza through this tube, which has 20 holes in the tip of it. This tube goes into the spinal fluid.”
Stewart continues, “It makes the injections so easy. It takes five to ten minutes to get the injection, and I’m able to do that sitting up in my wheelchair.”
Shawn says in a year and a half on Spinraza, his breathing and speech have improved.
And since doctor Muhonen’s Port simplifies drug delivery, he has high hopes for the future.
A company in Boston is working on improvements to doctor Muhonen’s Port right now.