A tablet formulation of Evrysdi (risdiplam) for spinal muscular atrophy (SMA) recently approved in the U.S. is designed to give patients a new option, a Genentech official said. “Evrysdi is the only oral, non-invasive disease-modifying SMA treatment, and the new tablet formulation may provide greater freedom and…

Treatment with Spinraza (nusinersen) can help stabilize motor function in adults with spinal muscular atrophy (SMA), according to a new analysis. “This study aligns with previously reported findings and suggests that long-term [treatment with Spinraza] offers sustained clinical benefits in adults with SMA,” researchers wrote in the abstract…

It’s been almost six years since the one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) was first approved in the U.S. to treat young children with spinal muscular atrophy (SMA). Now, more than 95% of babies diagnosed with SMA are being treated with Zolgensma, according to Daniel Grant, vice president…

When I close my eyes, I see the world as it should be — a place where accommodation isn’t something to fight for; where people with disabilities don’t have to plan every step, hoping the next doorway isn’t too narrow, the next curb isn’t too high, and the next space…

Now that I’ve been living in my new house for a month, I better understand what I love about it. In my last house, where we’d lived since I was 13, I had my own home within the home, which included my bedroom, bathroom, kitchen, and medical supply room.