Andrea Lobo,  —

Treatment with Evrysdi (risdiplam) is generally safe and effective at improving or stabilizing motor and lung function in adults with spinal muscular atrophy (SMA) for up to two years, according to a real-world study in Belgium. Results also suggested easing of difficulty swallowing and fatigue, as well as…

Interim results from a Phase 1 clinical trial show salanersen, Biogen’s treatment candidate for spinal muscular atrophy (SMA), slowed neurodegeneration, and improved motor function, in children with the genetic disease. The data also demonstrated that four of eight SMA children treated for at least one year attained new…

The case of a woman with spinal muscular atrophy (SMA) type 2 who developed an abnormal accumulation of molecules called ketone bodies was described in a recent case report in the U.S. Called ketoacidosis, this occurs when fats are broken down to obtain energy, resulting in ketone bodies being…

Training with a wearable robotic device called HAL, for hybrid assistive limb, improved walking ability for people with spinal muscular atrophy (SMA) who were also receiving treatment with Spinraza (nusinersen), according to a study in Japan. “[These] results may provide sufficient data to inform clinical decision-making about the…

SMA Europe will host its 1st International Clinical Care Symposium on SMA and focus on the biological causes of spinal muscular atrophy (SMA) and how to prevent and manage the symptoms that may lead to life-threatening emergencies. Titled, “Medical Emergencies in SMA: pathophysiology, prevention, and response,” the…

Treatment with Spinraza (nusinersen) and physical therapy that focused on posture improved motor skills in a young boy with spinal muscular atrophy (SMA) type 0 and prevented the progression of scoliosis. That’s according to a case report in Japan that demonstrated the boy was able to sit…

Most adolescents and adults with spinal muscular atrophy (SMA) have normal sensory nerve function, even those with long-lasting disease, regardless of SMA type, a study in the Netherlands shows. Nerve conduction tests had abnormal results in about 6% of the patients, which was about what was seen in healthy…

Poor sleep is associated with more severe depressive symptoms and respiratory issues in adults with spinal muscular atrophy (SMA), a survey study of 50 patients in Italy reports. Adequate sleep was more likely to be lacking and symptoms of depression were more severe in people with SMA type…

Newborn screening (NBS) programs for spinal muscular atrophy (SMA) were available in 33 countries as of the beginning of 2024, including the U.S. and most countries in the European Union. That’s according to a survey that gathered responses from experts in 80 countries. According to the researchers, “projections indicate…

Limited social interaction and difficulties in accessing needed medications and physical therapy were consequences of the COVID-19 pandemic, parents and grandparents of children with spinal muscular atrophy (SMA) in Sweden reported. In online surveys and chats, family members spoke of having to take charge of their child’s physiotherapy and…