2021
DOI: 10.1177/08830738211047019
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Health Profile of Preterm Males With Duchenne Muscular Dystrophy

Abstract: In this retrospective cohort study, we characterize the health profile of preterm males with Duchenne muscular dystrophy. Major clinical milestones (ambulation cessation, assisted ventilation use, and onset of left ventricular dysfunction) and corticosteroids use in males with Duchenne muscular dystrophy identified through a population-based surveillance system were analyzed using Kaplan-Meier survival curves and Cox proportional hazards modeling. The adjusted risk of receiving any respiratory intervention amo… Show more

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Cited by 2 publications
(2 citation statements)
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“…A CEA was undertaken to compare relative costs and effects of delandistrogene moxeparvovec plus SoC to SoC alone from a US healthcare system perspective using a simulated cohort of 4-year-olds with DMD [ 48 ]. SoC consisted of corticosteroids (prednisone/prednisolone), physical/occupational therapy, multidisciplinary assessments, and gastrointestinal/respiratory/cardiac management [ 11 ].…”
Section: Methodsmentioning
confidence: 99%
“…A CEA was undertaken to compare relative costs and effects of delandistrogene moxeparvovec plus SoC to SoC alone from a US healthcare system perspective using a simulated cohort of 4-year-olds with DMD [ 48 ]. SoC consisted of corticosteroids (prednisone/prednisolone), physical/occupational therapy, multidisciplinary assessments, and gastrointestinal/respiratory/cardiac management [ 11 ].…”
Section: Methodsmentioning
confidence: 99%
“…administration based on the patient’s body weight. With a median age at diagnosis of 4.6 years, 73 the average patient with DMD receiving gene therapy will be substantially heavier than a patient treated with onasemnogene abeparvovec, a gene therapy indicated for patients aged less than 2 years with SMA. 49 Another gene therapy approved in the US, voretigene neparvovec, is directly administered via subretinal injection.…”
Section: Case Study Of Delandistrogene Moxeparvovec a Gene Therapy Fo...mentioning
confidence: 99%