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 ...

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 ...

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

Individually and collectively, it’s vital to be sensitive to what’s happening in the world around us. This awareness allows ...

Since July, my family has been converting the garage space in our new house into my own bedroom, bathroom, kitchen, and medical supply room. We’ve faced one challenge after another as we prepare to ...