Infants with presymptomatic spinal muscular atrophy who were treated with risdiplam had improved functional and survival outcomes. At 12 months, most treated infants were able to sit and stand ...

The parents of two boys born with a rare genetic disorder have demanded the rollout of a life-changing heel prick test – ...

Oral risdiplam (Evrysdi, Genentech) started in the first 6 weeks of life let most infants with presymptomatic spinal muscular atrophy (SMA) reach motor milestones typical of healthy babies, results of ...

Biogen Inc. announced positive interim results from a Phase 1 study of salanersen (BIIB115/ION306), a novel antisense oligonucleotide for treating spinal muscular atrophy (SMA). The data show that ...

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 ...

SMA type 2, previously known as Dubowitz disease, is a hereditary disease that affects motor neurons. It typically becomes noticeable around the ages of 6 to 18 months, when children with the ...

The Health Union Review Board says that one in every 10,000 babies worldwide is born with spinal muscular atrophy, or SMA, a genetic and progressive neurodegenerative disease that affects the motor ...