Disease-modifying therapies (DMTs) may help lower the risk of children’s bone fractures among young patients with spinal muscular atrophy…
Articles by Patricia Inacio, PhD
Treatment with Spinraza (nusinersen), which was the first disease-modifying therapy to be approved for spinal muscular atrophy (SMA)…
Training with a wearable robotic hybrid assistive limb (HAL) device over two years was found to markedly improve the walking…
Newborn screening for spinal muscular atrophy (SMA) is now available to all babies born in Canada. The milestone announcement…
The prevalence of spinal muscular atrophy (SMA) at birth in the U.S. is lower than the historic global SMA…
Scholar Rock remains on track to report topline results later this year from its ongoing Phase 3 clinical trial…
Inhibiting an abnormally hyperactive protein called cyclin-dependent kinase 5 (Cdk5) significantly lessens the dysfunction and death of motor neurons, the…
A drop in the levels of four proteins in the cerebrospinal fluid (CSF) may be a marker of response to…
The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) safely and effectively treats infants with spinal muscular atrophy (SMA), especially…
Most of the infants and children with spinal muscular atrophy (SMA) in the RESPOND clinical trial who responded poorly…