Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children’s Research Hospital led the first in utero treatment of SMA with the orally administered drug risdiplam.
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children’s Research Hospital led the first in utero treatment of SMA with the orally administered drug risdiplam.
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