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 ...

A Glasgow man says he is “proud” that Scotland will begin screening new born babies for a rare genetic condition.

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 ...

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 ...

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 ...