In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene ...

A single dose of a new formulation of the gene replacement therapy intrathecal onasemnogene abeparvovec ( OAV101 IT) improved movement ability in teens and children older than 2 years with spinal ...

Spinal muscular atrophy (SMA) is a progressive genetic disease that causes devastating muscle atrophy and disease-related complications. SMA is the leading genetic cause of death in infants and ...

Scholar Rock’s spinal muscular atrophy (SMA) therapy has shown benefit in muscle function in children under 12 in a pivotal ...

Apitegromab met the primary endpoint in the SAPPHIRE trial, showing significant motor function improvement in SMA patients compared to placebo. Scholar Rock plans to submit a biologics license ...

Please provide your email address to receive an email when new articles are posted on . An investigational, fully human monoclonal antibody developed as a muscle-targeted therapy for spinal muscular ...

RESPOND study is actively enrolling, with baseline characteristics reporting infants and toddlers have residual unmet medical needs in multiple areas after gene therapy; SPINRAZA treatment following ...

A new study describes a base editing method for treating spinal muscular atrophy (SMA). This supports early signs that “single-shot” treatments from clinical trials using genome editing technologies ...

(MEMPHIS, Tenn. – February 19, 2025) Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children’s Research Hospital led the ...

Treatment with onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA) type 1 was associated with less need for nutritional support or nighttime ventilation compared to recommended ...