Most children SMA type 1 can survive an episode of pARDS, a life-threatening respiratory failure, under standard protocols, ...

"Our preliminary findings show that German children with SMA, despite significant physical disability, have surprisingly good subjective HRQoL." – Erik Landfeldt, MSc, PhD Previous studies have found ...

Adaptations that provide a balance between adventurous play and protective design for children with SMA include: Adaptive swings: Swings with high backs, secure harnesses, or molded bucket seats give ...

—A new qualitative participatory study explored how powered mobility (PM), in the form of modified ride-on cars, impacts children with spinal muscular atrophy type 1 (SMA1) and their families. Here ...

In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene ...

Please provide your email address to receive an email when new articles are posted on . At the time of last follow up, 82.6% of children born with SMA type 1 from 2016 on were still alive. Of the 140 ...

A multivariable analysis suggested the safety and efficacy of onasemnogene abeparvovec (OA) in the treatment of older and heavier children with spinal muscular atrophy (SMA) type 1. No serious ...

A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in children and adolescents with spinal muscular atrophy, according to a study ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

MADERA COUNTY, Calif. (KFSN) -- Like most one-year-olds, Damaris Perez spends a lot of time playing in her crib. The average person wouldn't know this baby has limited time left on earth. "It's been ...

First and only muscle-targeted SMA therapy to demonstrate statistically significant, clinically meaningful improvements on the gold-standard HFMSE scale (p=0.019) versus placebo, with consistent ...