Background: Therapeutic trials are critical to improving outcomes for individuals diagnosed with Duchenne muscular dystrophy (DMD). Understanding predictors of clinical trial participation could maximize enrollment. Methods: Data from six sites (Colorado, Iowa, Piedmont region North Carolina, South Carolina, Utah, and western New York) of the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) were analyzed. Clinical trial participation and individual-level clinical and sociodemographic characteristics were obtained from medical records for the 2000–2015 calendar years. County-level characteristics were determined from linkage of the most recent county of residence identified from medical records and publicly availa...
BACKGROUND: Duchenne muscular dystrophy (DMD) exhibits substantial variability in rates of disease p...
Background: Patients are the experts of their own experiences. In regulatory science, best practices...
Despite corticosteroids being the only treatment documented to improve strength and function in boys...
Introduction: Because of the variability in muscular dystrophy (MD) in terms of clinical manifestati...
BACKGROUND: Natural history data are essential for trial design in Duchenne (DMD) and Becker muscula...
International audienceBACKGROUND: To explore clinical heterogeneity of Duchenne muscular dystrophy (...
Background: Natural history data are essential for trial design in Duchenne (DMD) and Becker muscula...
Objective: With the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is...
Background: Recent short-term clinical trials in patients with Duchenne Muscular Dystrophy (DMD) hav...
BACKGROUND: Recent short-term clinical trials in patients with Duchenne Muscular Dystrophy (DMD) ha...
Background: Duchenne Muscular Dystrophy (DMD) is the most common muscular dystrophy, but it is a rar...
Introduction: Racial/ethnic differences in diagnostic and treatment services have been identified fo...
ObjectiveResearch has not examined the use of health care by patients with myotonic muscular dystrop...
Early clinical trials of therapies to treat Duchenne muscular dystrophy (DMD), a fatal genetic X-lin...
Objective: The aim of the study was to assess different outcome measures in a cohort of ambulant boy...
BACKGROUND: Duchenne muscular dystrophy (DMD) exhibits substantial variability in rates of disease p...
Background: Patients are the experts of their own experiences. In regulatory science, best practices...
Despite corticosteroids being the only treatment documented to improve strength and function in boys...
Introduction: Because of the variability in muscular dystrophy (MD) in terms of clinical manifestati...
BACKGROUND: Natural history data are essential for trial design in Duchenne (DMD) and Becker muscula...
International audienceBACKGROUND: To explore clinical heterogeneity of Duchenne muscular dystrophy (...
Background: Natural history data are essential for trial design in Duchenne (DMD) and Becker muscula...
Objective: With the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is...
Background: Recent short-term clinical trials in patients with Duchenne Muscular Dystrophy (DMD) hav...
BACKGROUND: Recent short-term clinical trials in patients with Duchenne Muscular Dystrophy (DMD) ha...
Background: Duchenne Muscular Dystrophy (DMD) is the most common muscular dystrophy, but it is a rar...
Introduction: Racial/ethnic differences in diagnostic and treatment services have been identified fo...
ObjectiveResearch has not examined the use of health care by patients with myotonic muscular dystrop...
Early clinical trials of therapies to treat Duchenne muscular dystrophy (DMD), a fatal genetic X-lin...
Objective: The aim of the study was to assess different outcome measures in a cohort of ambulant boy...
BACKGROUND: Duchenne muscular dystrophy (DMD) exhibits substantial variability in rates of disease p...
Background: Patients are the experts of their own experiences. In regulatory science, best practices...
Despite corticosteroids being the only treatment documented to improve strength and function in boys...