A unique mutation carried by an 8-year-old girl supported a diagnosis of spinal muscle atrophy with lower extremity ...

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 ...

Spinal muscular atrophy (SMA) is a genetic condition that causes muscle weakness and atrophy, which can affect your ability to take care of daily tasks. Occupational therapy (OT) can help with ...

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

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

Taldefgrobep alfa did not significantly improve motor function across the total population of treated spinal muscular atrophy (SMA) patients in a pivotal Phase 3 clinical trial, but significant ...

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