Various ethical and social factors influence care decisions for families affected by spinal muscular atrophy (SMA) in this new era of disease-modifying therapies (DMTs), according to a Canadian study. In interviews, caregivers reported that a range of factors, including financial resources and their expectations of treatment outcomes, influenced their…
News
Biogen has acquired Alcyone Therapeutics and will continue to develop ThecaFlex DRx, a device intended to ease patient experience and accessibility when delivering certain medicines into the spinal canal. The ThecaFlex DRx System is initially being evaluated with Spinraza (nusinersen) in people with spinal muscular atrophy (SMA),…
Measuring the levels of the survival motor neuron (SMN) protein in blood cells may help track spinal muscular atrophy (SMA) patients’ response to Evrysdi (risdiplam), a study from Japan found. Patients taking the drug showed a significant increase in blood levels of SMN after one month of therapy. SMN…
An experimental oral combination of two therapies eased motor symptoms and prolonged survival in a mouse model of spinal muscular atrophy (SMA), a study shows. The study, “The effect of coadministration of D156844 and AR42 (REC-2282) on the survival and motor phenotype of mice with spinal muscular atrophy,”…
Gene therapy GC101 safely improves or stabilizes motor function in children and adolescents with spinal muscular atrophy (SMA) types 2 and 3, a small study reports. Genecradle Therapeutics is also running three Phase 1/2 clinical trials to test the gene therapy in people with SMA type 1 (…
Called “Connecting the Dots,” a new awareness campaign from SMA Europe is highlighting the importance of transdisciplinary and breakthrough research in spinal muscular atrophy (SMA). The nonprofit umbrella organization behind the initiative, comprised of SMA advocacy and support groups from across Europe, said its aim is to…
Spinal surgery is often a major consideration for children and teens with spinal muscular atrophy (SMA) who develop scoliosis, a complication that can affect comfort, posture, and breathing. To better understand this step in SMA care, SMA News Today spoke with Arun Hariharan, MD, a pediatric orthopedic and spine…
Spinraza (nusinersen) helps correct a key amino acid deficiency in people with a severe form of spinal muscular atrophy (SMA), a study has found. Researchers found that the treatment restores levels of L-arginine, an amino acid important for brain development that is reduced in the brainstem and spinal…
Adults with spinal and bulbar muscular atrophy (SBMA) have elevated levels of neurofilament light (NfL) chain and glial fibrillary acidic protein (GFAP), two biomarkers for nerve damage, in the cerebrospinal fluid (CSF) that surrounds the brain and spinal cord, according to a study. Over two years, biomarker levels in…
Scholar Rock plans to have the the muscle-strengthening therapy apitegromab available commercially in the U.S. before the end of the year if the U.S. Food and Drug Administration (FDA) approves it next month as an add-on treatment for spinal muscular atrophy (SMA). The company sought…
