Scholar Rock announced that a Phase 3 clinical trial of its experimental muscle-targeting therapy apitegromab has finished enrolling children and young adults with spinal muscular atrophy (SMA) types 2 or 3, who are unable to walk but can sit independently. “Completing SAPPHIRE enrollment is a critical…
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The Muscular Dystrophy Association (MDA) has awarded one of 37 new research grants to a professor studying the role of survival motor neuron or SMN — the protein that’s missing in people with spinal muscular atrophy (SMA) — in disease development and treatment. Winning the three-year grant — in…
Biohaven Pharmaceuticals has completed the enrollment of children and young adults with spinal muscular atrophy (SMA) in a pivotal Phase 3 trial in the U.S. and Europe that is testing its experimental muscle-targeting therapy taldefgrobep alfa. The company had expected to recruit about 180 patients of all…
Children with spinal muscular atrophy (SMA) treated with Zolgensma (onasemnogene abeparvovec) used fewer healthcare resources and had lower medical costs than children on Spinraza (nusinersen), but they had higher SMA treatment costs, according to a recent U.S. analysis. Overall, healthcare costs for SMA patients using these disease-modifying…
In an African American toddler, neuromuscular symptoms of a severe form of nutritional rickets — a condition characterized by weak or soft bones due to dietary deficiencies — were initially mistaken as spinal muscular atrophy (SMA) type 3, according to a new case study. Researchers say the overlapping symptoms…
Adults with spinal muscular atrophy (SMA) experience more health problems affecting a range of bodily systems compared with the general population, according to a U.S. healthcare claims analysis. A total of 30 conditions examined in the study, many of which are associated with older age in the general population,…
Spinraza (nusinersen) led to clinically meaningful motor improvements for three of six adults with spinal muscular atrophy (SMA) types 2 or 3 who were treated at a hospital in Japan. Other aspects, including respiratory and swallowing function, did not change considerably. “The distinct efficacy of [Spinraza] requires further…
Note: This article was updated Sept. 25 to clarify that respiratory declines were observed in patients with SMA types 2 and 3 and that the type of blood clots observed in Evrysdi-treated patients were deep vein thromboses. Evrysdi (risdiplam) safely stabilizes or improves motor function for most people…
The European Commission (EC) has extended its approval of Evrysdi (risdiplam) to treat babies with spinal muscular atrophy (SMA) who are younger than 2 months old, making the therapy now available to treat SMA patients across all age ranges in Europe. Evrysdi was first approved in the European Union…
The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) safely and effectively treats infants with spinal muscular atrophy (SMA), especially when given within the first two months of life, according to the findings of a real-world, small study in Italy. Zolgensma “is an excellent therapeutic option for SMA patients” the…
