When I was born 31 years ago, spinal muscular atrophy (SMA) was a very different disease than it is today. There were no approved treatments. No gene therapy. No medication designed to slow its progression. For those of us born before the era of SMA treatments,…
Discussion
“What does your heart say?” Crouched next to the character of Kun, a newly freed deaf fish who lived trapped in an aquarium her entire life, was the personification of Water. Played by theater artists Jade Ow and Deborah Chai, respectively, on stage at Singapore’s Nanyang Technological University, the scene…
Discussion
A rapid, in-house genetic test allowed doctors in Japan to confirm a newborn’s diagnosis of spinal muscular atrophy (SMA) within hours and start treatment when the boy was 9 days old. The baby was readmitted to the hospital eight days after birth because of poor feeding and weight loss.
Discussion
Someone recently suggested that I write about my school days and what it was like growing up with spinal muscular atrophy (SMA). My initial reaction was to scrunch up my face and shrug, because my experience in school felt very normal to me, nothing especially remarkable or strange about…
Discussion
Children with spinal muscular atrophy (SMA) already living with symptoms experienced gains in motor abilities after receiving the gene therapy Zolgensma (onasemnogene abeparvovec-xioi), a small Brazilian study found. The real-world report tracked the outcomes of seven youngsters in the South American nation who were treated with Zolgensma only…
Discussion
Children and adults with spinal muscular atrophy (SMA), as well as their caregivers, may experience clinically relevant mental health symptoms, including anxiety and depression, a nationwide German study found. Among caregivers, those of patients with SMA type 1, the most common form of the disease, showed the greatest psychological…
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