The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to salanersen, a new, more potent Biogen treatment for spinal muscular atrophy (SMA) that’s now in late-stage testing. The treatment candidate, being evaluated in two ongoing Phase 3 clinical trials, is similar to — but…
Discussion
With SMA, I work hard to take advantage of every opportunity that comes my way. But what if I’m missing something? What if there are opportunities that are within my grasp, but I’m not aware of them? That’s why I’ve been doing some inner exploration. It can teach both…
Discussion
I’d be lying if I said I wouldn’t sell my soul for money. In a way, that’s what my job as a professional disability advocate and writer is: Mining my life as a resource for content to monetize, whether directly or indirectly. Is the net result adding good to…
A combination of genetic testing approaches enabled researchers in Spain to more accurately characterize complex genetic changes that may help explain differences in disease severity in spinal muscular atrophy (SMA), according to a new study. The findings suggest that “combining multiple genetic diagnostic techniques” enables more accurate genetic characterization…
Discussion
School is out in my area, and summer has officially started! Living with SMA means that my independence is made possible through a network of practical, daily support from friends in my community, and caregiving is woven into nearly every part of my life. My volunteer caregiving schedule…
Problems with swallowing, also called dysphagia, remain a concern for many children with spinal muscular atrophy (SMA) type 1 despite available treatments, a real-world study reports. Researchers examined swallowing in patients with SMA type 1 receiving disease-modifying therapies (DMTs). They found that…
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