Lily Sander will never forget what it was like to meet other people with neuromuscular diseases the first time she attended Muscular Dystrophy Association (MDA) Summer Camp. Living with Charcot-Marie-Tooth disease, Sander had met only one or two other people with neuromuscular diseases in her city. She was…
Discussion
Baby Jeffrey joined our family in mid-May 1997. Besides his abdominal breathing, alarming only my husband, Randy, he was perfect. Eight weeks later, my physician brother examined Jeffrey. Paul’s typically calming demeanor turned eerily somber after he found a dull-sounding lung and no reflexes. He felt confident our pediatrician would…
Discussion
Health-related quality of life for spinal muscular atrophy (SMA) patients treated with Spinraza (nusinersen) is closely linked to the well-being of their caregivers, particularly among patients who are able to sit, a South Korean study found. Participants with SMA who had poorer motor function generally reported lower quality…
Discussion
“Are you ready?” My caregiving friend stands beside my bed, poised to uncover me in the morning. I utter a very unladylike groan and clutch the edge of the warm blanket that is tucked under my chin. No, I’m not ready! The days are growing shorter and colder; the sun…
Discussion
Scholar Rock is working to bring spinal muscular atrophy (SMA) treatment Isembyld (apitegromab-mstn) to patients worldwide, following U.S. Food and Drug Administration (FDA) approval of the infusion therapy as the first muscle-strengthening agent to treat the disease. “Our work does not stop with the US approval,” Akshay…
Discussion
After its success in the U.S., Scholar Rock is once again seeking approval in the European Union for its muscle-strengthening drug apitegromab —now launching as Isembyld in America — for spinal muscular atrophy (SMA). The developer has resubmitted an application to the European Medicines Agency (EMA)…
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