Marta Figueiredo, PhD

Pediatric neurologists’ expectations in terms of treatment benefits are decidedly higher for infants and toddlers with spinal muscular atrophy…

Infants with spinal muscular atrophy (SMA) who are being screened for Zolgensma may present with maternal antibodies against the…

Evrysdi (risdiplam) was generally safe and well tolerated in a broader group of children and adults with spinal…

Zolgensma appears generally safe and well tolerated among spinal muscular atrophy (SMA) patients weighing 8.5 kilograms (about 18.7…

Treatment with Spinraza (nusinersen) appears safe in infants and children with spinal muscular atrophy (SMA) who responded poorly to the gene…

The availability of early genetic testing and approval of several spinal muscular atrophy (SMA) treatments has dramatically changed disease…

Treatment with Spinraza (nusinersen) stabilized or improved motor abilities in children and adults with spinal muscular atrophy (SMA) regardless…

Respiratory muscle strength is more severely affected in people with earlier-onset forms of spinal muscular atrophy (SMA), but it generally declines…

Blood biomarker levels of muscle damage — rather than nerve cell damage — are significantly altered in people with a…

Despite its well-reported benefits in motor function, Spinraza (nusinersen) may not be as effective at preventing or easing feeding and swallowing…