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.
The models and assays utilized in preclinical drug development play a crucial role in determining the success of clinical trials. However, findings from 2D cell […]
Pediatric neuromuscular diseases are genetically and clinically heterogeneous. Despite advances in diagnostic next generation sequencing, a substantial number of patients fail to be definitively diagnosed […]
The TREAT-NMD Advisory Committee for Therapeutics (TACT) is a unique multidisciplinary group of international experts including academic and industry drug development experts, representatives of patient foundations, […]
Increasing evidence implicates skeletal muscle as an active contributor to amyotrophic lateral sclerosis (ALS) pathology. Activin A and myostatin are potent negative regulators of skeletal […]
Background: Motor neuron disease (MND) is a progressive neurological disorder with no known cure that damages motor neurons. The purpose of this analysis is to […]
Background: Reliable and accurate assessment of motor function among children with rare neurologic disorders is critical for tracking disease progression and identifying a response to […]
Becker muscular dystrophy (Becker) is a serious, rare, neuromuscular disorder with no approved treatments. There is a need to understand the lived experience of Becker […]
BACKGROUND/OBJECTIVE: Emerging therapies targeting pathways involved in contraction-induced muscle injury have shown promising results in delaying disease progression for individuals with BMD. As novel therapies […]
Background: Cardiomyopathy is the leading cause of mortality for Becker Muscular Dystrophy (BMD). Risk stratification is challenging due to the wide variation of cardiomyopathy onset, […]