Community Perspectives

Whole-body quantitative MRI, or qMRI, can track long-term subtle muscle changes throughout the body among adults with spinal muscular atrophy (SMA), despite treatment and clinical stability, a new study reported. In fact, qMRI was seen…

My mother tried persuading me to get a hospital bed at home for years. “Your uncle can help us source one that the pediatric hospital doesn’t want,” she said at one point. “Our neighbor doesn’t need hers…

I was diagnosed with spinal muscular atrophy (SMA) in the mid-1980s at 18 months old. At the time, doctors explained to my parents that, because it is a genetic disease, there was a chance that they would…

Surgery to implant magnetically controlled growing rods (MCGRs) is a safe and effective option for children with spinal muscular atrophy (SMA) type 1 and scoliosis, according to a study. “MCGR represents a viable and…

“Spinal muscular atrophy golf tournament,” I spoke into my voice-to-text software. It was late at night, and I was physically drained. As a result, I knew my speech wouldn’t be very articulate. Nevertheless, I persisted in trying…

A home-based respiratory physiotherapy program focused on breathing exercises and airway clearance techniques can significantly improve quality of life for young people with spinal muscular atrophy (SMA) who are receiving treatment with Spinraza…

Abe Homer’s entry into healthcare gaming technology began at Children’s Hospital Colorado, where he met a young man with advanced spinal muscular atrophy (SMA). “He was a young man in his mid-20s, who was in a…

What is Spinraza for SMA? Spinraza (nusinersen) is an injection therapy widely approved for treating adults and children with spinal muscular atrophy (SMA). It was the first therapy approved to target the underlying causes of SMA. Mutations in the SMN1 gene, which contains instructions for cells to produce…

By the time I was 19, I believed I had escaped my reproductive cycle. In fact, I had developed a superiority complex about it. While my friends complained about cramps and cravings, I laughed in their…

Scholar Rock has resubmitted its application seeking approval in the U.S. of  its muscle-strengthening agent apitegromab — rejected last year due to manufacturing issues — for the treatment of children and adults with spinal…