Lung function in people with earlier-onset forms of spinal muscular atrophy (SMA) tends to decline gradually throughout childhood and stabilizes in early adulthood, according to data from a population-based natural history study. The study, “Natural history of lung function in spinal muscular atrophy,” was published in…
Although a disease of motor neurons, nerve cells of the spinal cord and brainstem, the earliest signs of spinal and bulbar muscular atrophy (SBMA) are in skeletal muscles, according to data from a mouse study. These findings suggest that changes in muscle strength and contraction are early disease…
The interplay between different types of nerve cells, including motor neurons and glial cells, seems to be at the root of motor neuron degeneration and denervation, or the loss of nerve supply, that characterizes spinal muscular atrophy (SMA), a review study reports. The study, “…
Spinal fusion surgery — a procedure to fuse two or more spine vertebrae — is more successful at reducing spine curvature in children with spinal muscular atrophy (SMA) type 1 and 2 if they have previously been given growth-friendly spinal implants, a study reports. But the…
Delays in diagnosing spinal muscular atrophy are still evident, particularly for those with SMA type 3, despite recent changes in recommendations for disease detection and care, a study from Italy reports. Such delays are particularly relevant as targeted therapies are now available, and growing evidence links greatest benefit to…
The first patient has been dosed in the Phase 2/3 DEVOTE trial assessing the safety, tolerability, and efficacy of a higher dose of Spinraza (nusinersen) in patients of all ages with spinal muscular atrophy (SMA), Biogen has announced. Spinraza, the first disease-modifying treatment approved by…
Young children given a single injection of Zolgensma into the spinal canal continue to show gains in motor function and safety, new interim data from the STRONG trial in spinal muscular atrophy (SMA) type 2 patients up to age 5 show. These findings from the open-label Phase 1/2 clinical…
The European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP), has recommended conditional approval of Zolgensma (onasemnogene abeparvovec) for the treatment of patients with spinal muscular atrophy (SMA) and a clinical diagnosis of type 1, or for those with SMA carrying up to three…
A single infusion of Zolgensma (onasemnogene abeparvovec-xioi) continues to lead to meaningful and sustained benefits in children with spinal muscular atrophy (SMA) treated in infancy, according to data from several clinical trials. Zolgensma, formerly known as AVXS-101, is a gene therapy originally developed by AveXis, now…
A gene called synaptotagmin 13 (SYT13) can prolong the lifespan and prevent the destruction of motor neurons in mouse models of amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA), according to a study. The study, “Synaptotagmin 13 is neuroprotective across motor neuron diseases,”…
