Treatment with OAV101 IT, a formulation of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) that’s delivered directly into the spinal canal, safely led to motor improvements for children with spinal muscular atrophy (SMA) type 2 who were at least 2 years old, top-line results from the Phase 3 STEER…
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Throughout 2024, SMA News Today brought our readers daily coverage of the latest clinical research and scientific breakthroughs related to spinal muscular atrophy (SMA). Here are the top 10 most-read stories we published last year, each with a brief description. We’re excited to continue serving the SMA community in…
Nearly 4 of every 5 infants with spinal muscular atrophy (SMA) type 1 born in Italy during the era of disease-modifying therapies (DMTs) are still alive, illustrating that such treatment has “radically changed the course of … type I SMA,” with “much higher survival,” according to a nationwide…
An algorithm that takes into account clinical measures of motor symptom severity alongside data on factors like age and height may help predict the risk of scoliosis in people with spinal muscular atrophy (SMA) who have received disease-modifying treatment. “We trained a scoliosis classifier, demonstrating how data give rise…
SMA Europe is launching an initiative to share personal stories of how treatment affects the lives of people with spinal muscular atrophy (SMA). The initiative comes as part of OdySMA, an SMA Europe project that tracks access to treatments and care for SMA patients across Europe, with…
Swallowing function may have improved in spinal muscular atrophy (SMA) patients since disease-modifying therapies (DMT) have become available, a review study suggests. The analysis showed problems with swallowing were prevalent in the years before the emergence of DMTs and that studies after the treatments became available indicated stabilizations…
Gratitude and support from family and healthcare staff can help people with spinal muscular atrophy (SMA) feel more positive, stay motivated, and plan their lives better, a study found. Programs that focus on gratitude could help patients improve their mental health and help them cope with their disease more…
Evrysdi (risdiplam) effectively improved motor function in adults with spinal muscular atrophy (SMA) type 2 or type 3, particularly skills like hand strength and function, a small study in France shows. Most patients also reported gains in bulbar functions, that is, speaking, chewing, and swallowing, and eased breath…
Treatment with the approved gene therapy Zolgensma was found to ease breathing difficulties in children with spinal muscle atrophy (SMA), reducing hospitalizations due to respiratory issues by than 25%, according to a real-world study in Israel. Daily ventilation time — the period when some children used mechanical devices to…
Researchers reported a unique mutation that supported an 8-year-old girl’s diagnosis of spinal muscle atrophy with lower extremity predominance (SMA-LED). The mutation, mapped to the BICD2 gene, resulted in a severe form of SMA-LED, type 2B, that emerged before birth and was primarily marked by joint contractures in infancy.
