After five years of daily treatment with Evrysdi (risdiplam), 90% of children with spinal muscular atrophy (SMA) type 1 were alive, and most had reached motor milestones, such as sitting without support, that are rarely seen in untreated children with this severe disease. These are the final published…
Discussion
Daily treatment with Evrysdi (risdiplam) may help stabilize or improve motor function and improve well-being in adults with severe forms of spinal muscular atrophy (SMA), according to a real-world study from the Netherlands. The study showed Evrysdi may benefit people with longstanding SMA …
Discussion
When my great-aunt was 100, she used to sing a song that said “count your many blessings.” As I approach another birthday and complete another year on my journey with SMA, I’m counting my blessings one by one. I’ve had plenty of crises — or as I call them,…
Discussion
Muscle ultrasound helps assess muscle health and its association with motor function in children with spinal muscular atrophy (SMA) treated with Spinraza (nusinersen), according to a recent study. While the findings varied by muscle group and SMA type, ultrasound data generally indicated that children with SMA exhibited muscle wasting…
Discussion
One of the queerest ways to spend Pride Month could go like this … You decide to go to the theater one night. You scan the ticketing site for a show and land on one about lesbian identity, struggles, and culture in Singapore. It’d be no fun to go alone,…
Discussion
The one-time gene therapy Itvisma (onasemnogene abeparvovec-brve) is now approved in the European Union for people with spinal muscular atrophy (SMA) ages 2 and older — a regulatory decision that will make the injection therapy available to SMA patients of all ages. According to therapy developer Novartis,…
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