Spinraza treatment may help restore lipid balance to the cerebrospinal fluid (CSF) of children with SMA, according to a small ...

SMA newborn screening programs were available in 31 countries in 2023, covering about 7% of the world's newborns, per a study.

A mother was given SMA treatment Evrysdi during pregnancy, and her child with SMA, now 2.5, has not shown any signs of the ...

A study found profound swallowing deficits in SMA type 1 babies, a disease feature of note as disease-modifying treatment ...

The U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of Evrysdi (risdiplam), an approved oral ...

A UK study found more than 80% of 80 SMA type 1 children on a DMT had scoliosis or other abnormal spinal curvature needing ...

A Valentine's card exchanged reminded columnist Halsey Blocher that disabled people also deserve opportunities to serve others.

Treatment with Spinraza (nusinersen), which was the first disease-modifying therapy to be approved for spinal muscular atrophy (SMA) back in 2016, does not lead to significant kidney dysfunction in ...

It was another appointment in an appointment-packed January. My hopes weren’t particularly high, but no one will ever accuse me of being a less-than-perfect patient, so I braved the cold bite of the ...