Which research institution led the first in-utero treatment for Spinal Muscular Atrophy (SMA)?
St. Jude Children’s Research Hospital
Why is the news?
“Scientists Achieve First In-Utero Treatment for Spinal Muscular Atrophy Using Risdiplam”
St. Jude Children’s Research Hospital led the first in-utero treatment for Spinal Muscular Atrophy (SMA) using the oral drug risdiplam. This breakthrough offers hope for early intervention in SMA, a neurodegenerative disorder that begins affecting individuals before birth.
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