On a perfectly mundane February afternoon, my mom and I drove to downtown St. Paul, Minnesota. It’s a new ritual now that I’ve transitioned most of my care to Gillette Children’s Specialty Healthcare. Every six months or so, I spend the afternoon at their adult clinic, where I am…
Spinal muscular atrophy (SMA) is associated with multi-organ changes in a protective protein modification that helps guard cells against molecular damage, according to a study in mice. The researchers also found that treatment with antisense oligonucleotides — the same type of SMN-targeting approach used in Spinraza…
Low levels of SMN protein in muscle stem cells (MuSC) — specialized cells that help sustain muscle growth and regeneration — may contribute to the loss of motor neurons, the nerve cells that control movement and are progressively lost in spinal muscular atrophy (SMA), a new study in mice…
Discussion
There aren’t many guarantees in life, but if you live in New England, you can always be sure there will be a winter nor’easter. With these storms come snow, wind, and power outages. The local news outlets remind everyone to get their bread and milk before the shelves are cleared,…
Discussion
I’m a lot of people’s favorite something. That’s not bragging, it’s just a statement of fact. Ask my mother who her favorite child is, or some of the nurses at my usual hospital’s neurology ward who their favorite patient is, or some of my friends who their favorite friend or…
Treatment with Evrysdi (risdiplam) may modestly improve arm function for adults with spinal muscular atrophy (SMA), a new study from the Czech Republic and Slovakia indicates. Findings suggest that adults with SMA generally have stable motor and lung function over three years of Evrysdi treatment. Evrysdi “was associated…
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