News

Adjustments add up to more comfort in new wheelchair

Michael, my patient who has spinal muscular atrophy (SMA), has recently transferred into a new wheelchair and was experiencing significant discomfort in his right hip while seated. We performed several different modalities and activities on Michael’s tailbone to increase his comfort. A combination of these adjustments was…

Consistent DMT access is major concern for caregivers in Canada

Consistent access to disease-modifying therapies (DMTs) is a major concern for parent caregivers of children with spinal muscular atrophy (SMA) in Canada, a study reported. Barriers to such treatments, as described by caregivers, included variable regulatory approvals across different Canadian provinces, high costs, and insufficient healthcare infrastructure. “Our…

Oral SBMA therapy shows safety and tolerability in Phase 1 trial

NIDO-361, Nido Biosciences’ experimental oral treatment for spinal and bulbar muscular atrophy (SBMA), was seen to be generally safe and well tolerated in healthy adults who participated in an initial clinical trial. Results of the Phase 1 study (ACTRN12622000964718) were presented during the 2023 International Kennedy’s…

2 type 1 SMA babies show signs of restored motor neuron connections

Treatment with Spinraza (nusinersen) and Zolgensma (onasemnogene abeparvovec) led to increases in the electrical activity of motor neurons — the specialized nerve cells that control voluntary movement — for two young children with type 1 spinal muscular atrophy (SMA), according to a new report. Findings from tests…