Author Archives: Andrea Lobo

A home-based respiratory physiotherapy program focused on breathing exercises and airway clearance techniques can significantly improve quality of life for young people with spinal muscular atrophy (SMA) who are receiving treatment with Spinraza…

Children with spinal muscular atrophy (SMA) showed trends suggesting that nutritional status may be linked to motor function, according to a study in Poland that tracked 38 children over one year. Researchers found…

Even when treated early with disease-modifying therapies (DMTs), infants and toddlers with spinal muscular atrophy (SMA) do not develop swallowing skills at the same pace or to the same level as healthy children. That’s according…

Genentech will not advance emugrobart (GYM329) into Phase 3 development, after early results from a clinical trial testing it in combination with Evrysdi (risdiplam) in people with spinal muscular atrophy (SMA). According to a…

Treatment with Spinraza (nusinersen) led to sustained improvements in motor function in adults with spinal muscular atrophy (SMA), and showed a manageable safety profile, according to a nationwide study in South Korea. Among 19 adults, researchers…

In children with spinal muscular atrophy (SMA) type 1, the most severe form of the genetic disease, treatment with Spinraza (nusinersen) restored levels of taurine — an amino acid that plays a…

Low levels of SMN protein in muscle stem cells (MuSC) — specialized cells that help sustain muscle growth and regeneration — may contribute to the loss of motor neurons, the nerve cells that control movement and…

The blood creatinine-to-cystatin-c ratio (CCR), an indicator of muscle mass and strength, may be a biomarker of treatment response in children with spinal muscular atrophy (SMA), a study in China shows. Increases in the ratio were significantly…

The strength of nerve signals to muscles may serve as a marker of disease severity, and as a means to track responses to Spinraza (nusinersen) treatment, in children with spinal muscular atrophy (SMA), the…

The case of a 51-year-old man ultimately diagnosed with spinal muscular atrophy (SMA) type 4 is believed to mark the first time this form of the rare genetic disease has had as its main…