The first patient has been dosed in a clinical trial testing GB221, a next-generation gene therapy for spinal muscular atrophy (SMA) type 1. “I would like to express our gratitude for the young child and family…
One of my rules is that nurses cannot care for me when they’re sick. Although this creates challenges, I don’t want to risk exposure. Because I live with SMA, the repercussions of a virus or other cause of…
Countless events shape the course of our lives: marriage, career changes, parenthood, the loss of loved ones, and health crises, to name just a few. The list is almost endless. After eight years of marriage, my…
I love to volunteer. If I see a need that I can meet, I am eager to jump in. And if someone asks me to help with a project or service, I am quick to say…
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…
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 —…
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…
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…
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…
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…
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