SMA children

A method that uses laser light to get information from inside the body in a noninvasive way may help monitor how spinal muscular atrophy (SMA) is progressing in real time and how children are responding to therapy, a study suggests. Signals from optoacoustic imaging, which can picture how…

A harness system designed for children with movement difficulties, the Portable Mobility Aid for Children (PUMA) improved or stabilized motor function among young patients treated for spinal muscular atrophy (SMA) in a small study. More frequent at-home use of the system, whose harness supports a child’s…

The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) didn’t cause any unexpected safety issues among children with spinal muscular atrophy (SMA) who weighed up to 21 kg (about 46 pounds) in a Phase 3b clinical trial, and most had stable or improved motor function a year after treatment.

The case of a 4.5-year-old girl with spinal muscular atrophy (SMA) type 2 who had acute pain and swelling in her legs after physiotherapy due to multiple fractures was recently described in India. The child had poor bone health, but the fractures were considered…

  Despite concerns about long-term efficacy and safety, gene therapy was the first choice treatment for most parents of children with spinal muscular atrophy (SMA), whether diagnosed via newborn genetic screening or symptoms, according to a new survey study. However, the amount and type of information parents received upon…

Children with spinal muscular atrophy (SMA) generally have poor bone health, including low bone mineral density and reduced bone formation and remodeling, a study indicates. The effect on bone health is more pronounced in children with worse motor function, but as treatment with Spinraza (nusinersen) improved motor function…