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…
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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…
August is Spinal Muscular Atrophy (SMA) Awareness Month where community members will join together on behalf of patient advocacy and research funding to help find a cure for the rare genetic condition. “August is our time to shine a brighter spotlight on the needs, wants, hopes, and experiences of…
Most parents and potential parents in the U.K. support screening newborns for spinal muscular atrophy (SMA) as part of the National Health Service (NHS) Newborn Screening Programme, according to a survey developed and funded by Novartis. “We believe that this government has an opportunity to make newborn screening…
A New York-based scientist has been awarded for his pioneering work that led to the development of Spinraza (nusinersen), the first RNA-based therapy to target the underlying cause of spinal muscular atrophy (SMA). Adrian R. Krainer, PhD, a professor at Cold Spring Harbor Laboratory, in New York, will formally…
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…
A novel mutation in the ASAH1 gene was identified as the cause of spinal muscular atrophy with progressive myoclonic epilepsy (SMA-PME) in a 4-year-old boy, according to a recent report. Despite the genetically confirmed diagnosis, the boy showed no signs of myoclonic seizures, or brief muscle spasms, which…
