The U.S. Food and Drug Administration (FDA) has extended its review of risdiplam as an oral treatment for spinal muscular atrophy (SMA) by three months, pushing the decision due date to August 24 from May 24. This extension was based on additional clinical data submitted by Roche — one…
A specific chemical modification of antisense oligonucleotides (ASOs) — called MOE; used in Spinraza (nusinersen) — was more effective than another commonly used modification — called PMO — in improving survival and motor neuron preservation in a mouse model of severe spinal muscular atrophy (SMA). These findings…
Higher levels of free-fat body mass and lean body mass are associated with better motor function in children with spinal muscular atrophy (SMA) types 1 and 2, a study from researchers in Italy found. The findings highlight the importance of monitoring SMA patients’ nutrition, and suggest that these…
Risdiplam treatment has led to “clinically meaningful” improvements — whether gains or disease stabilization — in the broadest range of spinal muscular atrophy (SMA) patients, by age and disability level, yet enrolled in a clinical trial. More than one-third of those in the second part of the SUNFISH trial…
Risdiplam safely and significantly improved or stabilized motor function in children and young adults with spinal muscular atrophy (SMA) types 2 and 3, one-year data from the second part of the SUNFISH trial show. As anticipated by risdiplam’s developers, these benefits were more pronounced in younger patients. “Risdiplam is the…
Researchers in China report having developed a method that was fast, effective, and reliable in newborn screening (NBS) for spinal muscular atrophy (SMA). The results, “Newborn Screening for Spinal Muscular Atrophy in China Using DNA Mass Spectrometry,” were published in the journal Frontiers in…
Analysis of the protein levels in the cerebrospinal fluid (CSF) of people with late-onset spinal muscular atrophy (SMA) may help to identify biomarkers that not only monitor but also predict Spinraza responses, a study suggests. The results of the study, “Cerebrospinal fluid proteomic profiling…
Increasing the amount of ZPR1 — a protein found in low levels in people with spinal muscular atrophy (SMA) — improved motor function and extended the lifespan of a mouse model of SMA, a study reports. The data, which also identified the protein as a direct regulator of the…
SMA News Today brought you daily coverage of key findings, treatment developments — including in-depth coverage of Zolgensma’s approval in the U.S. — and of clinical trials related to spinal muscular atrophy (SMA) throughout 2019. We look forward to reporting more of this relevant news to patients, family members, and…
Spinraza (nusinersen) safely stabilizes or improves motor function in older patients with spinal muscular atrophy (SMA), according to a small U.S. real-world study. Researchers also pointed out that objective measures of meaningful improvements in daily life activities are required to better determine treatment effectiveness in this patient population.
