In a groundbreaking medical achievement, doctors have successfully treated a life-threatening motor neuron disease in a baby ...

The mother took the drug daily for six weeks while the baby herself was given the drug orally, roughly one week after the ...

An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...

Spinal muscular atrophy, a progressive neurodegenerative disorder set in motion before birth, could be treated in utero, a ...

This study demonstrates the critical role of Afadin on the generation and maintenance of complex cellular layers in the mouse retina. The data are solid, which provides important insights into how ...

Based on alignment gained with FDA under the Regenerative Medicine Advanced Therapy (RMAT) designation, Neurona plans to conduct the Phase 3 ...

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude ...

Discover the first in-utero treatment for spinal muscular atrophy (SMA) using risdiplam. Promising results show no SMA signs ...

February 19, 2025) Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists ...

But the performance of the technology—how easily and accurately a BCI user’s thoughts move a cursor, for example—is limited ...