Spinal muscular atrophy (SMA) is an inherited condition that affects motor neurons, which are the nerve cells that control the movement of voluntary muscles. Their loss leads to progressive muscle weakness. Fatigue in SMA Fatigue is a state of physical exhaustion. It can decrease motivation and affect everyday activities,…
Spinal muscular atrophy (SMA) is a rare genetic condition characterized by the progressive loss of motor neurons, which are the specialized nerve cells that control voluntary movement, leading to muscle weakness and atrophy, among other symptoms. There are several types of SMA, based on the affected gene, age…
Novartis Pharmaceuticals‘ investigational compound BVS857 significantly improved muscle volume in patients with X-linked type spinal muscular atrophy (SMA) over a short period of time, Phase 2 clinical results show. However, the compound failed to improve muscle strength and function. These findings were reported in the study, “…
Blood levels of a waste product from muscle metabolism could be used to see how spinal and bulbar muscular atrophy (SBMA) develops before symptoms appear, a Japanese study reports. The research on the waste product, creatinine, appeared in the journal Neurology. The title of the…
Levels of a male hormone produced by the adrenal glands could provide useful information on the severity of spinal and bulbar muscular atrophy, or SBMA, a study reports. The identified the hormone as DHEA sulfate. Researchers’ study, published in the Journal of Neurology, is titled “The metabolic…
The patent application for gene therapy candidate INT41, developed by Vybion for the treatment of Huntington’s disease, spinal muscular atrophy (SMA), and other neurodegenerative diseases, was granted Track One status from the U.S. Patent and Trademark Office (USPTO). The patent application covers the treatment’s composition and methods of use.
Patients with spinal and bulbar muscular atrophy (SBMA) may be at risk of developing respiratory complications due to a decline in the levels of genes responsible for muscle strength, according to a new study. The authors assert that monitoring the breathing decline in these patients may help provide better clinical…
Difficulties with adequate nutrition and feeding persist among children and adolescents with spinal muscular atrophy (SMA) types 2 and 3 being treated with Spinraza (nusinersen), a Norwegian study reports. Important nutrients, such as protein, fiber, vitamins, and minerals, often were consumed in lower-than-recommended amounts by these patients, while…
Despite its well-reported benefits in motor function, Spinraza (nusinersen) may not be as effective at preventing or easing feeding and swallowing difficulties in children with spinal muscular atrophy (SMA) type 1, according to a single-center study in the U.K. Those with SMA type 1c, this type’s least severe subgroup, were more…
The Canadian Agency for Drugs and Technologies in Health (CADTH) will not recommend reimbursement for Zolgensma in children with spinal muscular atrophy (SMA) who are more than 6 months old, citing insufficient evidence of efficacy and safety in older patients. “Other countries are providing guidance and funding based…