Columns

I did something that I’m afraid to publicly admit. Sure, I did it willingly. And in this corner of the internet, I share my life openly and honestly with those who are willing to read it. So it only seems fair that, despite my hesitancy to share this with…

Any parent who has received a spinal muscular atrophy (SMA) diagnosis for their child can tell you that early treatment is crucial. Children who receive treatment for SMA as soon as possible are more likely to achieve important motor milestones. One-time gene therapy ZOLGENSMA® (onasemnogene abeparvovec-xioi) can stop…

Damn Taylor Swift and her lyricism. I had a different plan for this column, with a different title in mind for it. I was going to ramble about moving out of the condominium I’ve been staying in for almost two years. There was to be a farewell to its…

Up to 4.5 years of Spinraza (nusinersen) treatment led to meaningful improvements in motor function in adolescents and adults with spinal muscular atrophy (SMA), according to a medical records analysis. “[Spinraza] was effective in long-term follow-up,” researchers wrote. Noting a dearth of data on Spinraza’s long-term effectiveness…

Spinraza (nusinersen) continues to show sustained efficacy in improving motor function in infants and toddlers with spinal muscular atrophy (SMA), according to newly released interim data from the SHINE study. The greatest benefits were among children who began being treated with Spinraza before they were 10 months old.

Working with the biotech company Genentech never ceases to amaze me. Back in 2019, when Genentech representatives first reached out to me about an upcoming initiative, I had no idea what to expect. They wanted me to attend a meeting, along with several other people in the…

A delayed diagnosis in spinal muscular atrophy (SMA) may occur in many cases due to the absence of clinical suspicion of the rare genetic disorder, an interview-based study concluded. In particular, treating physicians of pediatric patients often disregarded or misinterpreted signs reported by parents, leading to diagnostic delays,…

Children with spinal muscular atrophy (SMA) who have lower levels of an inflammatory protein called interleukin-8 (IL-8) in the fluid surrounding the brain and spinal cord tend to respond better to treatment with Spinraza (nusinersen), a new study suggests. The findings indicate that IL-8 is a better predictor…

“Your 60s you’ll have a major surgery, the music is still loud but it doesn’t matter because you can’t hear it anyway. Seventies, you and the wife retire to Fort Lauderdale, you start eating dinner at 2 o’clock in the afternoon, you have lunch around 10, breakfast the night…

After two years of tweaking, my new wheelchair is home for good. I think. Most wheelchair users dread new devices. There’s the process of submitting the claim to insurance, which is frankly straight from hell. Then there’s the seating — a monthslong process that involves strangers studying your…