Please provide your email address to receive an email when new articles are posted on . A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its ...

Spinal muscular atrophy with respiratory distress (SMARD) is a rare genetic condition that typically affects infants and children. It causes muscle weakness and breathing problems. Spinal muscular ...

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 ...

Spinraza, also known as nusinersen, is a medicine used to treat spinal muscular atrophy (SMA) in children and adults. SMA is a genetic disease that is passed down through families. It leads to muscle ...

Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 currently approved disease-modifying therapies that restore SMN protein ...

Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. So most recently, there has been the ...

The FDA approved a risdiplam tablet for SMA patients aged 2+ and over 44 lbs, matching the oral solution's efficacy and safety. Risdiplam, an SMN2 pre-mRNA splicing modifier, enhances SMN protein ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

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 ...

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 ...

Evrysdi, also known as risdiplam, is an oral medicine used to treat spinal muscular atrophy (SMA) in children and adults. SMA is a genetic disease that is passed down through families. It leads to ...