On the Hunt for the Next Breakthrough in Motor Neuron Disease
The Scientist Magazine,
Researchers leave no stone unturned in the search for a spinal muscular atrophy treatment strategy that uses base editing.
Researchers leave no stone unturned in the search for a spinal muscular atrophy treatment strategy that uses base editing.
SMA Type 1, the most common and most severe, develops in babies less than 6 months old.
A new study has laid out the steps that connect the neuromuscular disorder spinal muscular atrophy (SMA) to the mutations in…
Roche is moving into spinal muscular atrophy SMA with risdiplam, which could gain FDA approval by May.