Shutterstock | SciePro
Lenten Campaign 2025
This content is free of charge, as are all our articles.
Support us with a donation that is tax-deductible and enable us to continue to reach millions of readers.
A recent case study in The New England Journal of Medicine describes a medical breakthrough in which a group of US doctors treated an unborn baby with a rare genetic condition while still in utero. The unborn child had tested positive for type 1 spinal muscular atrophy (SMA), a rapidly progressing disease that severely weakens the muscles, making it impossible to move or breathe. Children with this condition have historically died by their second birthday. 
The pregnant woman in the case study had lost her first child to the terrible disease and was willing to work with doctors in an experiment in which she received the drug Risdiplam every day for six weeks before giving birth. 
Risdiplam works by increasing the concentration of a crucial protein called survival motor neuron (SMN) protein in order to prevent the break down of nerve cells in the brain and body; such proteins are lacking in those with the genetic mutations that cause SMA. The drug had previously only been approved by the FDA to treat infants ages 2 months and older. The FDA made a special, one-time exception for this case study. 
During the six weeks that the expectant mother took the medication, testing found that the medicine passed through her umbilical cord and into the amniotic fluid surrounding her baby.
After birth, the infant continued to receive the oral medication daily, and the regimen has proven successful thus far. Pediatric neurologist Michelle Farrar, a high-profile SMA researcher in Australia, told Smriti Mallapaty at Nature that the baby girl “has been effectively treated, with no manifestations of the condition” even 30 months after birth.
Aleteia reached out to Kelly Mantoan, the Catholic author of Better Than OK: Finding Joy as a Special-Needs Parent. Mantoan is a mother of five; two of her children have SMA and they take Risdiplam, which has slowed the progression of their disease. She is also the founder of Accepting the Gift, a Ministry for Catholic Special Needs Parents. 
Here’s what she had to say about this amazing medical breakthrough: 
“When my sons were diagnosed with SMA, there were no FDA approved treatments. But today there are three, including Risdiplam. SMA is no longer presented to parents as a death sentence but a condition that can be treated, saving the lives of countless unborn children with SMA and improving the quality of life for children and adults with the condition. It feels like the miracle mothers like myself prayed for for years.”
To read more about this remarkable medical achievement, check out The New England Journal of Medicine.
Get Aleteia delivered to your inbox. It’s free!
Articles like these are sponsored free for every Catholic through the support of generous readers just like you. Please make a tax-deductible donation today!
Help us continue to bring the Gospel to people everywhere through uplifting Catholic news, stories, spirituality, and more.
Get Aleteia delivered to your inbox. It’s free!

source