Panelists discuss how the needs of older patients with spinal muscular atrophy (SMA) differ from infants, focusing on ...

Panelists discuss how spinal muscular atrophy (SMA) progresses over time, affecting motor function differently in older ...

The therapy used molecules called antisense oligonucleotides, or ASOs, that can alter the expression of genes through interactions with RNA, which creates proteins. ASOs are currently given to babies ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec.

The scientists at the University of California, San Francisco, and Johns Hopkins University in Baltimore injected an ...

Injecting medicine into the amniotic fluid staves off progression of spinal muscular atrophy in utero.Evidence is mounting ...

Scientists at Northwestern University have developed a new approach that directly combats the progression of ...

Ridhay’s parents received the devastating diagnosis just months ago, and they’ve since been living each day with a renewed ...

Digital PCR to simultaneously detect SMN1 gene defects and the number of SMN2 copies to diagnose SMA matches standard methods ...

and visible cramps or twitching from lower motor neuron degeneration Bulbar involvement (caused by damaged nerve cells in ...

will present preclinical data supporting the potential of its tRNA therapy in Duchenne Muscular Dystrophy (DMD). The study demonstrated that Tevard’s suppressor tRNA rescued full-length dystrophin ...