My younger brother, Kevan, and I were both diagnosed at a young age with SMA, so we grew up with power wheelchairs and powerful imaginations. We lived most of our childhood in our own pretend play world, where we could be spies, horse farmers, and baseball players; we…
Children with spinal muscular atrophy (SMA) often struggle to get a good night’s sleep — and their caregivers also commonly report feeling sleep-deprived, according to a new study. “Sleep problems have potentially critical consequences for children and are also to a great extent related to caregivers’ sleep. We therefore…
Children with spinal muscular atrophy (SMA) who start on a disease-modifying therapy (DMT) — four have been approved in the last decade — before developing symptoms are usually able to feed by mouth, but SMA patients who already have swallowing difficulties before beginning treatment often continue to have…
Discussion
I am honored to have the opportunity to share my journey as a mom to an extraordinary human being. My son, Brandon Johnson, has faced immeasurable odds and lives a life that reflects resiliency and strength of spirit. In my professional life, over the past 46 years, I have been…
The blood creatinine-to-cystatin-c ratio (CCR), an indicator of muscle mass and strength, may be a biomarker of treatment response in children with spinal muscular atrophy (SMA), a study in China shows. Increases in the ratio were significantly associated with gains in motor function following long-term treatment with Spinraza (nusinersen). Although…
Discussion
“Do you remember when Dad took us kite flying?” I asked my brother, Gabriel. We were on a train ride home from the Sands Theatre, where we’d just seen an international production of Disney’s live musical, “Beauty and the Beast.” “Nope,” he said. “I thought that was with the…
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