October 17th 2025
At CNS 2025, Kathryn Xixis, MD, highlighted the growing adoption of EPAs and outcomes-based learning in pediatric neurology.
Boy presents with fatigue, minimal responsivity, and diffuse muscle weakness
August 7th 2024An 11-year-old boy with a history of asthma and allergic rhinitis presented to the emergency department (ED) with worsening fatigue, minimal responsivity to external stimuli, and diffuse muscle weakness for 2 months.
Treatment candidate for limp-girdle muscular dystrophy receives Rare Pediatric Designation
June 18th 2024The company also announced that it has surpassed its interim analysis enrollment target and expects topline phase 3 data from the FORTIFY study (NCT05775848) among individuals with LGMD21/R9 in 2025.