disease-modifying therapy

Children with SMA express good quality of life in small study

Children with spinal muscular atrophy (SMA), even those with substantial physical disability, tend to report that their quality of life is similar to other children their age, a small study from Germany reports. “The results from this pilot study show that German children with SMA, despite significant physical disability,…

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…