Andrea Lobo,  —

An investigational gene therapy that targets nerve cells thats’s being developed by Exegenesis Bio was well tolerated and improved motor function in a mouse model of spinal muscular atrophy (SMA), a study shows. Based on the data, EXG001-307 seems to have a better safety profile than the approved…

Biogen is very pleased with the “striking” results seen thus far in clinical testing with its therapy candidate salanersen — designed to have high efficacy in people with spinal muscular atrophy (SMA) — and is engaging with health authorities on the design of further trials that might provide…

A higher-dose regimen of nusinersen, currently up for approval in the U.S., may provide further benefits to people with spinal muscular atrophy (SMA) relative to the approved Spinraza regimen, according to its developer, Biogen. Nusinersen is marketed as Spinraza. The Phase 3 DEVOTE clinical trial (NCT04089566) part C showed…

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…