Note: This column describes the author’s own experiences with fentanyl and other pain medications. Not everyone will have the same response to treatment. Consult your doctor before starting or stopping a therapy. “You really know pain, huh?” someone asked me, eyes wide with concern. In response to their offering me…
Spinraza (nusinersen) is able to help slow lung function decline in people with spinal muscular atrophy (SMA), even if treatment is started when patients are already experiencing disease symptoms, a new study shows. The beneficial effects of the approved SMA therapy on lung function were generally more pronounced…
People with spinal muscular atrophy (SMA) show high long-term adherence to treatment with Evrysdi (risdiplam), with several patients remaining on the oral therapy after three years, according to a real-world study in Spain. The observational study, which involved adults and children with different SMA types, demonstrated that all…
Discussion
My aunt, who doesn’t throw anything away, recently sent me an artifact from my childhood: a pink paper pamphlet titled “Meet Connie.” I was diagnosed with SMA type 2 when I was 18 months old and started using a power wheelchair when I was in first grade. Around the…
The use of disease-modifying therapies (DMTs) — all widely approved in the last 10 years — may help children with spinal muscular atrophy (SMA) maintain nutrition and prevent stunted growth, an Australian study suggests. Children with SMA type 1 and type 2 who received approved DMTs…
Treatment with Spinraza (nusinersen) was linked to measurable changes in a range of cerebrospinal fluid (CSF) biomarkers in people with spinal muscular atrophy (SMA), some of which were associated with improvements in motor function, according to a review of published studies. Among the protein changes seen in CSF,…
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