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 (…
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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…
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…
A man with type 3 spinal muscular atrophy (SMA) and no history of diabetes developed diabetic ketoacidosis, a severe diabetes complication, which researchers said may be linked to metabolic changes. SMA can cause major loss of muscle, which plays a key role in processing sugars and fats. Muscle loss…
An infant with spinal muscular atrophy (SMA) type 1 and neutropenia was successfully treated with with Zolgensma (onasemnogene abeparvovec-xioi), according to a case report. Neutropenia, which refers to low levels of immune cells called neutrophils, increases the risk for infections. However, the child “tolerated the treatment well and,…
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…
