The posters will rotate daily. Please use the letter in the poster number to determine which day they will be exhibited.
Late Breaking posters are indicated by titles preceded by LB. Please search “LB” in the Title/Keywords to view all Late Breaking abstracts.
Background: Duchenne muscular dystrophy (DMD) is an X-linked muscle-wasting disorder characterized by muscle weakness, cardiac arrhythmia, and respiratory weakness due to the mutations in the […]
Background: Patients with spinal muscular atrophy (SMA) often require invasive ventilatory support (typically tracheostomy). While onasemnogene abeparvovec (OA), a one-time gene therapy, has demonstrated safety […]
Background: FA is an autosomal recessive neurodegenerative disorder that gradually impairs coordination, balance, and mobility. Traditional clinical assessments of FA are subjective and may encounter […]
The mdx mouse model of Duchenne muscular dystrophy (DMD) exhibits pathophysiological responses to graded forms of psychological stress, with phenotypes ranging from transient hypotension and […]
Background: Human induced pluripotent stem cells (iPSCs) have revolutionized biomedical and translational research in many fields such as neuromuscular diseases, cardiovascular diseases, development, aging, regeneration, […]
Background: Onasemnogene abeparvovec (OA), a one-time, adeno-associated-virus–based gene replacement therapy, has established efficacy for treatment of spinal muscular atrophy (SMA), a rare genetic neuromuscular disease. […]
Although Duchenne Muscular Dystrophy (DMD) is a progressive and degenerative disease there is evidence for embryonic and fetal stage defects during myogenesis, including transcriptional and […]
Background and objectives: Omaveloxolone (SKYCLARYS®) is approved for the treatment of Friedreich ataxia in patients 16 years of age and older in the United States. […]
Prenatal testing and newborn screening can promptly identify at-risk infants for SMA, but treatment may be delayed due to procurement complexities. The FDA expanded risdiplam […]