Guest Voice: Hussein’s journey is symbolic of our hope for peace
Our journey began long before my son, Hussein, was born. Due to a family history of SMA, my husband underwent genetic testing to be sure he was not a carrier. We received news that he…
Columns
New high-dose Spinraza regimen may offer added benefits in SMA
A new high-dose regimen of Spinraza (nusinersen) was recently approved in the U.S. as a treatment for spinal muscular atrophy (SMA), with clinical trial data showing a safety profile broadly consistent with the original 12…
Global study shows how a country’s wealth affects SMA diagnosis rates
Spinal muscular atrophy (SMA) is more common in wealthier countries across the globe, likely reflecting increased access to diagnostic tools, newborn screening, and modern treatments. That higher prevalence — the total proportion of a population…
What does it mean to be sexy with a disability like SMA?
Unintentionally, last year, most of my immediate family bought me Christmas gifts with the same theme. My brother, Gabriel, got me a handmade blue-and-white scarf from China. Mom got me a sheer-ish white shawl. My…
The ‘plinks’ in a life — the small ones and the big ones
On a recent Saturday morning, the dryer wouldn’t turn on. The washer held a big load of washed towels. As our recently adopted dog, Ellie, was still in unexpected heat, laundry duty was…
How my wheelchair fits into new friendships
There are many disabilities out there that significantly affect people’s lives but are not immediately noticeable to the general public. That is not the case with spinal muscular atrophy (SMA). As an adult living with SMA type 2,…
Expanding my SMA care team with mental health support
The Apple TV series “Shrinking” features an ensemble of messy characters. Lead protagonist Jimmy (played by Jason Segel) is a widower who struggles to care for his teenage daughter, Alice (Lukita Maxwell). As a therapist, he…
Guest Voice: Embracing the beauty of imperfection as an artist with SMA
My parents were the first to notice that I had some art talent. Spinal muscular atrophy (SMA) wasn’t well understood when I was a child, and in an era before iPads, cellphones, and travel…
SMA treatment Itvisma gets European regulatory panel OK
A European Medicines Agency (EMA) panel recommended that the one-time gene therapy Itvisma (onasemnogene abeparvovec-brve) be approved for patients with spinal muscular atrophy (SMA) ages 2 and older. The Committee for Medicinal Products for Human Use (CHMP)…
Newborn SMA screening widely supported in U.K. survey
Newborn screening (NBS) for spinal muscular atrophy (SMA) is widely supported among families whose babies were screened, SMA families and adults living with the disease, healthcare professionals, and the public, according to a large U.K. survey. The potential…
Recent Posts
- Guest Voice: Hussein’s journey is symbolic of our hope for peace
- New high-dose Spinraza regimen may offer added benefits in SMA
- Global study shows how a country’s wealth affects SMA diagnosis rates
- What does it mean to be sexy with a disability like SMA?
- The ‘plinks’ in a life — the small ones and the big ones