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Most spinal muscular atrophy (SMA) patients with four or more copies of the backup SMN2 gene achieved or maintained motor milestones after treatment with Zolgensma (onasemnogene abeparvovec), according to real-world data from the RESTORE patient registry. The one-time gene therapy from Novartis was also found safe in…

It’s been nearly two weeks since most of the U.S. “sprang forward” into daylight saving time (DST), and no matter how many times we go through the biannual time changes, it always seems to take this long for everyone to fully adjust. Some people may even still need to…

Something you may not know about me is that I have two homes. My first abode is a one-story home that was built to fit my needs almost 20 years ago. It’s in a quiet neighborhood with broken-down streets that make going out for a walk difficult in my…

Treatment with apitegromab for two years continues to stabilize or improve measures of motor function, fatigue, and life quality in children and young adults with spinal muscular atrophy types 2 and 3 who are not able to walk. That’s according to data from the TOPAZ clinical…

Children with spinal muscular atrophy (SMA) given Zolgensma (onasemnogene abeparvovec-xioi) gene therapy in clinical trials are maintaining, years later, the motor milestones they achieved in the original studies — and some have hit additional milestones even without further treatment. That’s according to data from long-term follow-up (LTFU)…

My hands are shaking as I look at my crowdfunding page and type. My mouth is dry and my heart is pounding. I feel like an overheated device running too many apps at once, except the apps are emotions. Anxiety tops the list. For half a year, I’ve…

Motor function is still stable or improved among people with spinal muscular atrophy (SMA) types 2 and 3 after four years of treatment with Evrysdi (risdiplam), according to updated results from the SUNFISH clinical trial. Patients and caregivers also reported continuous improvement or stabilization in the level…

If it’s not my mom rocking out to “The Final Countdown” by Europe, it’s my dad reminiscing about all the cartoons we used to watch. Shows like “Arthur” and “Dragon Tales” were mainstays in the Albers household. But my favorite was “Cyberchase,” an after-school program about a bunch of…

“OK, SMA. You win,” I thought after signing off from a virtual meeting. I didn’t know how it was possible to feel so defeated yet relieved about finding a solution for one of my recent health woes. But there I was, staring up at my ceiling and arguing…

Older children with spinal muscular atrophy (SMA) type 2 or 3, ages 2–5, given a single injection of Zolgensma into the spinal canal, showed clinically meaningful gains in motor function after one year, according to final data from the STRONG study. Such gains also were significantly better…