In spinal muscular atrophy (SMA), the death of nerve cells — which results in the progressive muscle weakness and wasting that characterize the rare disease — begins in wire-like nerve fibers known as axons, a new study from researchers in Scotland shows. The findings suggest that current SMA…
Discussion
There are often movies from our adolescent years that we continue rewatching no matter how old we get. They feel a bit like being wrapped up in a cozy blanket. I was definitely a Disney kid, so for me, “Camp Rock” and its sequel easily make that list,…
Discussion
A generous neighbor recently urged me to pick tomatoes from his garden during his extended absence. Last Thursday, I obliged. Helen Baldwin plays “sorta farmer” after picking tomatoes at a neighbor’s farm. (Photo by Helen Baldwin) I shared some with a friend whose own tomatoes were a…
Discussion
Evrysdi (risdiplam) may improve nighttime breathing in adults with spinal muscular atrophy (SMA), a small, real-world study in Italy found. After 12 months on Evrysdi, study participants experienced fewer episodes of breathing that stopped or became too shallow during sleep, along with fewer associated drops in blood oxygen…
Discussion
There are moments when I forget that I have spinal muscular atrophy (SMA). Does that surprise you? It surprises me. It’s not as if this is a new diagnosis that I’m still adjusting to. I’ve lived with this disability for as long as I can remember, and…
Discussion
Scholar Rock has successfully removed a manufacturing facility from its application to the U.S. Food and Drug Administration (FDA) seeking approval of apitegromab, an experimental muscle-strengthening treatment for spinal muscular atrophy (SMA). The move follows an announcement earlier this month that the company planned to remove…
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