Andrea Lobo,  —

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…

Nonadherence of spinal muscular atrophy (SMA) patients to their prescribed regimen of Evrysdi (risdiplam) — an oral solution generally taken once daily after a meal — is associated with higher healthcare costs, particularly in patients with type 2 disease. That’s according to a real-world study in the…

Blood levels of creatinine, a byproduct of normal muscle function, may be a potential biomarker of disease severity and treatment response in adults and adolescents with spinal muscular atrophy (SMA), a study in China shows. Creatinine levels, overall, strongly associated with disease severity across two years of follow-up, and…

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…

Young children with severe spinal muscular atrophy (SMA) have substantial loss of motor neurons in the thoracic region of the spinal cord, the area between the neck and the bottom of the ribs, a study found. The study provides a detailed look at SMA pathology in the spinal cord,…