
Gene therapy improves motor function in spinal muscular atrophy patients
Key Takeaway:
A single dose of onasemnogene abeparvovec significantly improves motor function in untreated spinal muscular atrophy patients, offering a promising new treatment option for this life-threatening condition.
What this means for you
This promising treatment for spinal muscular atrophy is not yet available in clinics. It's important to continue your current care and discuss any questions with your doctor.
Citation:
Nature Medicine - AI Section, 2025. Read article →



