Designation is supported by an exploratory analysis from the Phase 1b study showing that some children with SMA previously treated with gene therapy who had suboptimal clinical status experienced ...

Long-term data from DEVOTE/ONWARD studies show benefits of high dose nusinersen in people living with spinal muscular atrophy (SMA) New Phase 1b data further illustrate potential of salanersen in SMA, ...

A trial of Biogen’s spinal muscular atrophy (SMA) therapy Spinraza (nusinersen) suggests that a higher dose provides benefits to a broad range of patients—including those who have and have not been ...

Biogen Inc. (NASDAQ:BIIB) on Wednesday announced in a press release the topline results from the Phase 1 study of salanersen (BIIB115/ION306), an antisense oligonucleotide (ASO) being developed for ...

Biogen is running a campaign aimed at spreading awareness of the next-gen version of its spinal muscular atrophy therapy Spinraza. The FDA first approved Spinraza (nusinersen) back in 2016, making it ...

Spinraza is approved in more than 71 countries to treat infants, children, and adults with spinal muscular atrophy. The study achieved a statistically significant improvement in motor function in ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

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 ...

Injections for spinal muscular atrophy (SMA) refer to two treatments that the Food and Drug Administration (FDA) has approved for SMA: nusinersen (Spinraza) and onasemnogene abeparovec-xioi (Zolgensma ...

Adult-onset spinal muscular atrophy (SMA) is a rare neuromuscular disorder that primarily affects the motor neurons in the spinal cord, leading to muscle weakness and atrophy. SMA is part of a group ...

Spinal muscular atrophy (SMA) type 0 is a rare genetic disorder that affects the ability to control muscle movement. It’s present before birth, and infants born with the condition will need immediate ...