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 ...

"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 ...

Researchers evaluated oxygen saturation and arterial CO2 variables to screen for sleep disordered breathing in children with SMA type 1-3. Of the measures evaluated, only an oxygen desaturation index ...

KANSAS CITY, Mo. (KCTV) - One resilient metro baby is thriving after a rare genetic disorder diagnosis.

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

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 ...

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 ...

Monday, Novartis AG (NYSE:NVS) presented new data for Zolgensma (onasemnogene abeparvovec), a one-time gene therapy for the treatment of spinal muscular atrophy (SMA). Final data from the SMART Phase ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Motor function and other motor milestones were ...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today presented positive results from the primary analysis of the ongoing RAINBOWFISH ...

Parents Speak Out After Motorists Target Son with Disabilities at School Bus Stop School Transportation News Wire Reports ...

Positive CHMP opinion is based on data from the DEVOTE study which evaluated the high dose regimen of nusinersen in treatment-naive participants ...