News

The Committee for Medicinal Products for Human Use (CHMP) favors European Union (EU) approval of Evrysdi (risdiplam) being extended to cover babies with spinal muscular atrophy (SMA) younger than 2 months of age. The recommendation now will be reviewed by the European Commission, which is expected to issue…

Measuring levels of neurofilament light chain (NfL) in the blood could be used to detect subtle nerve damage that may occur as a side effect of experimental treatments for diseases like spinal muscular atrophy (SMA). That’s according to the study, “Neurofilament Light Chain: A Translational Safety Biomarker…

Children with spinal muscular atrophy (SMA) experience impaired health-related quality of life even when using disease-modifying therapies, according to a recent Swedish study. Still, over time on treatment, some patients experienced life quality gains, particularly those with SMA type 2. These improvements were largely related to improved…

Nonambulatory young adults with spinal muscular atrophy (SMA) who have contractures  — when muscle and connective tissue around a joint become short and hardened — do not feel significantly hindered by these in their daily lives. Despite contractures in multiple joints, they do not perceive them as a constant…

Decreased levels of the survival motor neuron (SMN) protein — missing or present in low levels in spinal muscular atrophy (SMA) — can cause defects in the structure and function of a brain’s cerebellum. This can result in difficulties with motor control and affect the ability to coordinate…

Children diagnosed with spinal muscular atrophy (SMA) and started on Spinraza (nusinersen) as presymptomatic newborns safely continue to achieve motor milestones with no need for permanent ventilation after five years of treatment, updated data from the ongoing NURTURE trial show. For the majority of trial children with…

A histone deacetylase (HDAC) enzyme inhibitor called AR42 was found to prolong survival and ease motor symptoms in a mouse model of spinal muscular atrophy (SMA). While scientists initially expected the experimental molecule to show benefits by helping to boost levels of the SMN protein that’s deficient in…

Most of the infants and children with spinal muscular atrophy (SMA) in the RESPOND clinical trial who responded poorly to the gene therapy Zolgensma showed gains in motor function with Biogen’s Spinraza (nusinersen), according to early results. Prior preclinical studies have suggested Zolgensma may target…

A growing number of young children with spinal muscular atrophy (SMA) type 1 who started taking Evrysdi (risdiplam) as babies are gaining the ability to sit, stand, and walk, according to four-year data from the FIREFISH clinical trial. No new deaths have been reported since the study’s…

Add-on treatment with Scholar Rock‘s muscle-targeted therapy apitegromab continues to stabilize or improve motor function for young spinal muscular atrophy (SMA) types 2 and 3 patients who entered the study unable to walk, according to a three-year update from the TOPAZ clinical trial and its…