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A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…
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I’m about to give myself credit where credit is due: I didn’t freak out for several months. I have undiagnosed, but undeniable, health anxiety. So, I was surprised by my reaction to a recall announced last June by Philips Respironics of 13 respiratory machines, including the BiPAP machine I’ve…
After being treated with Evrysdi (risdiplam) for at least a year, pre-symptomatic infants with spinal muscular atrophy (SMA) have retained the ability to swallow, and most have been able to stand and walk within developmentally normal windows. That’s according to new data from the RAINBOWFISH clinical trial presented…
My family and I have just returned home from a fun-filled, relaxing vacation in Orlando, Florida. We haven’t taken a vacation in over two years, and it was long overdue. Vacations offer opportunities to explore a different part of the world and take a break from normal routines. But sometimes…
Biogen is continuing to actively recruit patients for its Phase 4 RESPOND trial, which is evaluating the benefits of Spinraza (nusinersen) in infants and children with spinal muscular atrophy (SMA) who responded poorly to the gene therapy Zolgensma. Participants are now being enrolled at 10 sites in the U.S. and one in Italy,…
While I can’t speak on behalf of every medical professional I’ve come across in my lifetime (truth be told, that’d be quite a large number), I wouldn’t be surprised if they thought I didn’t own many clothes. I wouldn’t blame them. After all, I’ve been wearing the exact same pair…
“Hey, is that Lego?” my brother asks, a spark of interest in his eyes. He leans over to get a better view of my laptop. He’s 17, but in that moment, I see the 7-year-old boy who loved Lego so much, he’d want to immediately begin work on every set…
Children with spinal muscular atrophy (SMA) type 2 or 3 in China have lower bone mineral density — putting them at increased risk for bone fractures — compared with healthy children of the same age and sex, a study has found. Furthermore, SMA type 2 children had significantly…
Columnist Brianna Albers discovers a remake of her favorite video game is forthcoming, but she worries that it won’t be accessible.
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Becoming educated and informed about spinal muscular atrophy is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your SMA journey.
There is no cure for SMA yet, but there are treatments that can help manage the disease. Learn more about current therapeutic approaches here.
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