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 ...

Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

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 ...

Please provide your email address to receive an email when new articles are posted on . At 5 years, 91% of pediatric patients treated with Evrysdi were alive, 81% without permanent ventilation. Data ...

"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 ...

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 ...

The FIREFISH study showed that most children with type I spinal muscular atrophy (SMA) maintained or improved motor functions and feeding abilities over 5 years when treated with risdiplam. At the ...

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 ...

Novartis' Zolgensma is approved in over 51 countries, and more than 3,700 patients have been treated globally across clinical trials. Most patients in the SMART study maintained motor milestones ...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new 5-year data confirming the sustained efficacy and safety profile ...

Medical researchers have uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that overcomes important ...