Switch to Zolgensma From Evrysdi Found Safe, Effective for Baby Girl

The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) was safe and effective in a baby girl with spinal muscular atrophy (SMA) type 1 who was previously on Evrysdi (risdiplam) for about three months, a case study reports. This likely represents the first report of safety and efficacy outcomes of…

Spreading the Word

Spreading awareness about SMA has been one of our goals since we first were given Ella’s diagnosis in 2011. We immediately started a blog for our friends and family, so that important updates could be disseminated in one place rather than having to repeat…