OBJECTIVE: To compare outcomes by steroid treatment among non-ambulatory DMD patients. BACKGROUND: Corticosteroids are the standard of care for DMD; however, steroid use varies after […]
Background: Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder caused by a lack of functional dystrophin. Ataluren promotes readthrough of an in-frame premature stop […]
Background: Strategic Targeting of Registries and International Database of Excellence (STRIDE) Registry (NCT02369731) is an ongoing, multicenter, observational registry providing data on ataluren use in […]
Background: Strategic Targeting of Registries and International Database of Excellence (STRIDE) Registry (NCT02369731) is an ongoing, multicenter, observational registry providing data on ataluren use in […]
Background: Strategic Targeting of Registries and International Database of Excellence (STRIDE) Registry (NCT02369731) is an ongoing, multicenter, observational registry providing data on ataluren use in […]
Objective: Describe changes in outcomes (healthcare conditions, services, costs, and care days) in FSHD patients vs matched controls (MCs) two-years post versus pre diagnosis. Background: […]
Background Changes associated with the COVID-19 pandemic include a rise in demand for nursing services with an increase in turnover. The impact on outpatient operations […]
The evolutionarily conserved Notch signaling pathway is vital for the development and maintenance of multiple tissues. In skeletal muscle, the Notch pathway is involved in […]
ACTA1 encodes skeletal muscle-specific alpha-actin, which polymerizes to form the thin filament of the sarcomere, and mutations in ACTA1 are responsible for roughly 30% of […]