It’s been three years since COVID-19 lockdowns were implemented around the world. When that happened, time seemed to grind to a halt as the world intently followed the latest public health announcements. Plans were ripped apart as businesses all around us were shuttered. Three years later, as we’re still…
Note: This column describes the author’s own experiences with Evrysdi. Not everyone will have the same response to treatment. Consult your doctor before starting or stopping a therapy. I never thought it would be this difficult to secure a life-altering treatment. This is about my best friend and…
Children and adolescents in New Zealand with spinal muscular atrophy (SMA) will be able to access Evrysdi (risdiplan) through the country’s publicly funded healthcare system, starting in May. The decision by New Zealand’s Pharmaceutical Management Agency, known as Pharmac, means eligible patients will now have access to two…
Regular readers of my column may know that I love Taylor Swift. I’ve written multiple columns explaining how her songwriting has given me solace. I still buy physical copies of her albums (even though my laptop has no CD drive), and I’ve attended her concerts each time she’s visited my…
A single gene-editing treatment that converts the SMN2 gene into a functioning copy of SMN1 increased levels of the SMN protein that is lacking in spinal muscular atrophy (SMA), bringing better motor function to a mouse model of the disease, a study reported. Combined with Spinraza (nusinersen), an…
The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) can improve motor outcomes for children with spinal muscular atrophy (SMA) effectively, especially when it’s given in the first months of life, a review paper shows. “There is substantial evidence of improved outcomes when Zolgensma is administered early to children under…
I’m just a normal guy who happens to have physical limitations because of SMA. At least, that’s how I want others to see me. But is that how I see myself? I ask because I can be extremely hard on myself, sometimes even getting on my own nerves. Some…
Evrysdi (risdiplam) may be associated with longer survival and more motor function gains than Spinraza (nusinersen) in children with spinal muscular atrophy (SMA) type 1, according to an indirect comparison of clinical trial data. Sponsored by Roche — which markets Evrysdi — the analysis “leverages the longest…
Children with spinal muscular atrophy (SMA) given Zolgensma (onasemnogene abeparvovec-xioi) gene therapy in clinical trials are maintaining, years later, the motor milestones they achieved in the original studies — and some have hit additional milestones even without further treatment. That’s according to data from long-term follow-up (LTFU) studies…
My hands are shaking as I look at my crowdfunding page and type. My mouth is dry and my heart is pounding. I feel like an overheated device running too many apps at once, except the apps are emotions. Anxiety tops the list. For half a year, I’ve known…