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The U.S. Food and Drug Administration (FDA) has given NMD Pharma the green light to conduct a Phase 2 trial of its oral therapy NMD670 in people with spinal muscular atrophy (SMA) type 3. The upcoming clinical trial aims to evaluate the efficacy, safety, and tolerability…

I once knocked over our Christmas tree when I was 3 years old. To this day, I’m not proud of it, and frankly, I’m still a little traumatized by it. I was simply trying to get a good view out the bay window to see our…

My parents used to joke that I should’ve been born an American. As a kid, I loved burgers and fries, I was enamored with Disney movies and country music, and I spoke “proper” English better than anyone else in my family. That’s not to say I didn’t love it…

There are more than 1,200 people with spinal muscular atrophy (SMA) living in Italy, and the vast majority are on disease-modifying treatment, according to a new study. SMA types 2 and 3 are the most common forms of the disease in the country, but the…

The use of long-term breathing help, or ventilation, has markedly increased among children with spinal muscular atrophy (SMA) type 1 and other conditions since 2008, according to a survey of ventilation centers in the U.K. This increase coincided with the expanded use of various types of non-invasive masks…

The other day, my friends and I were talking about our mantras for the upcoming year. I usually start thinking about mine sometime in December, so this conversation was par for the course — something about the darkest month of the year makes me yearn for the optimism…

UNITY Screen, a noninvasive prenatal blood test, can provide an accurate personalized risk assessment for hereditary diseases such as spinal muscular atrophy (SMA) in fetuses, according to a recent study. Offered by BillionToOne in the U.S., the single-gene noninvasive prenatal test (sgNIPT) included in UNITY Screen…

The elevated production of the cell-death-related protein c-Fos was closely linked to the death of motor neurons in two mouse models of spinal muscular atrophy (SMA), a study showed. Motor neurons are the muscle-controlling nerve cells that are lost in people with SMA. Although c-Fos didn’t directly cause…

I listened to 78,978 minutes of music and podcasts in 2022. Thanks to the popular feature Spotify Wrapped, I and millions of users can look at a summary of our listening habits each year, for better or worse. Some may find my avid consumption of pop albums…

This year has been unprecedentedly good in terms of media. From season four of “Stranger Things” to the delightfully campy “Our Flag Means Death,” I’ve had no shortage of things to binge. But the highlight has undoubtedly been the renewed interest in “Star Wars.” It started with the six-episode…