News flash: First-ever prenatal treatment for SMA shows promise

In a first-of-its-kind approach, a mother at 32 weeks pregnant began taking Evrysdi, an oral SMA therapy that boosts SMN protein levels, and started her baby on Evrysdi shortly after birth; now, at 2.5 years old, the child shows no SMA symptoms, marking a groundbreaking development in the fight against one of the most severe genetic disorders. The parents in this case were both known carriers of SMA genetic variants and had a prior infant born with SMA-1 before current treatments became available.

👥 Who It’s For
Evrysdi is approved for SMA at all ages, but in this instance was used prenatally for known carrier parents with multidisciplinary oversight, although prenatal use remains unapproved.

🔬 How the Treatment Works
Evrysdi raises SMN protein levels, can cross the placenta, and may protect motor neurons before birth when administered to pregnant mothers.

📈 Success So Far
At 2.5 years old, the child shows no SMA-related symptoms and no treatment-linked adverse effects, with other minor health issues likely unrelated.

❤️ What This Means for You
This early intervention approach could offer new hope for families at risk, suggesting the possibility of preventing SMA symptoms with further validation.

⏳ What’s Next?
More clinical trials, regulatory reviews, and informed genetic counseling will guide safe and effective prenatal use of Evrysdi in high-risk cases.

📣 Voices of Support
Richard Finkel, MD, Director of the Center for Experimental Neurotherapeutics at St. Jude Research Hospital:
“The results suggest it would be worthwhile to continue investigating the use of prenatal intervention for SMA identified in utero.”

View the full SMA News Today article here

Read more about this development:

Independent: Independent covers the promising new SMA treatment approach, detailing its potential to change prenatal care.

Nature: Nature explores the scientific implications of the prenatal SMA treatment and its groundbreaking potential.

St. Jude Children’s Research Hospital: St. Jude highlights their involvement in advancing SMA treatments and their ongoing research efforts.

PharmaPhorum: PharmaPhorum discusses how this new approach could change the landscape of genetic disorder treatments.

Learn more about SMA with this guide

The report is available for free online on the AllMyHealth website.

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