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,…
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…
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.
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…
“You must have dropped the baby.” That sentence was one of the many knives flung at my mother’s chest when I was diagnosed with SMA. She was 24, barely an adult. I was a…
Three months ago, my husband, Randy, and I adopted Ellie, a mama dog who had been at the rescue for four months. She arrived with six puppies, tending to another litter after hers were adopted. “Tending” might…
Evidence-based guidelines have been developed for newborn screening (NBS), diagnosis, and early management of spinal muscular atrophy (SMA) in Australia and New Zealand to help ensure that all affected children are identified and treated as early as…
Caregivers are an essential aspect of my life with SMA, because I need assistance to do most of my activities of daily living. But I am learning more and more that caregiving…
Children with spinal muscular atrophy (SMA) who were diagnosed through newborn screening and treated early had better long-term independence and daily functioning, especially when supported by multidisciplinary care at home and at school, according to…
The U.S. Food and Drug Administration (FDA) is expected to decide in the coming months whether or not to approve apitegromab, an experimental muscle-strengthening agent designed to improve motor function in people with…
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