News

Nerve proteins may serve as biomarkers for SBMA progression

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…

Study shows SMA infants with neutropenia can take Zolgensma

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,…

Higher-dose nusinersen may further improve SMA outcomes: Developer

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…