Gene activity in SMA may be dysregulated, but the way the disease affects genes seems to vary across types of tissues, per a ...

Children with severe SMA have substantial loss of motor and sensory neurons in the thoracic region of the spinal cord, a ...

Liver-specific SMN1 gene therapy both improved liver functioning, and also reduced other SMA-like symptoms in a mouse model ...

Taldefgrobep alfa as an add-on therapy significantly improved motor function in pivotal Phase 3 trial, but racial differences ...

Years after losing a child to SMA, columnist Helen Baldwin shares that she has many blessings to be thankful for — courtesy ...

More than half of SMA patients in an Australian survey said they did not notice any improvement in their symptoms following ...

Recent progress in SMA research and advocacy was celebrated at Cure SMA's annual Hope on the Hill event in Washington, D.C.

SMA treatment Spinraza followed by Zolgensma improved motor function and prevented worsening of breathing difficulties in a ...

Since her SMA assignment, columnist Helen Baldwin has learned to look for the bright spots — even in Hurricane Helene's ...