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 pretty advanced stage of SMA at that point, where he could only…
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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 muscular atrophy (SMA). The drug developer said it expects the U.S. Food and…
The U.S. Food and Drug Administration (FDA) has approved a high-dose regimen of Spinraza (nusinersen) — one that allows patients to receive consistently higher doses of the therapy after a shorter initial treatment loading phase — for spinal muscular atrophy (SMA). The new dosing regimen is authorized for use…
Children with spinal muscular atrophy (SMA) showed trends suggesting that nutritional status may be linked to motor function, according to a study in Poland that tracked 38 children over one year. Researchers found that increases in BMI — a measure…
A combination of approved treatments, alongside early physical therapy, was successfully deployed to help treat a boy with spinal muscular atrophy (SMA) type 0 in Japan. Type 0 is the most severe form of SMA, marked by symptoms that start in the womb prior to birth. Without…
Even when treated early with disease-modifying therapies (DMTs), infants and toddlers with spinal muscular atrophy (SMA) do not develop swallowing skills at the same pace or to the same level as healthy children. That’s according to a new study from Germany, which tracked more than 30 children with…
Genentech will not advance emugrobart (GYM329) into Phase 3 development, after early results from a clinical trial testing it in combination with Evrysdi (risdiplam) in people with spinal muscular atrophy (SMA). According to a letter to the SMA community, this “difficult decision” follows results from the first…
In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To…
The motor function benefits of Itvisma (onasemnogene abeparvovec-brve), one of two Novartis’ gene therapies for spinal muscular atrophy (SMA), continued to increase over more than one year of treatment, extended Phase 3 trial results show. These findings come from the STEER Phase 3 trial (NCT05089656), which supported…
Up to three years of treatment with Evrysdi (risdiplam) appears safe and was associated with presymptomatic children with spinal muscular atrophy (SMA) reaching and maintaining key developmental milestones, according to Phase 2 study results. “The majority of children treated with [Evrysdi] before the onset of SMA symptoms, maintained…
