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

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 ...

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 ...

Novartis said new interim data from an ongoing phase clinical trial for its $2.1 million-per-patient gene therapy Zolgensma showed spinal muscular atrophy patients experienced significant therapeutic ...

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 ...