The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to the mother during pregnancy and to the child after birth. More than 2 years ...

(MEMPHIS, Tenn. – February 19, 2025) Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children’s Research Hospital led the ...

Optic atrophy is a degenerative disease of the optic nerve that leads to gradual damage to the retinal ganglion cells—the nerve cells that transmit visual signals from the retina to the brain. The ...

Optic atrophy is a degenerative disease of the optic nerve that leads to gradual damage to the retinal ganglion cells - the nerve cells that transmit visual signals from the retina to the brain. The ...