June 18th 2025
Many parents said they were afraid of noninvasive ventilation, with some citing inaccurate concerns about ventilation devices.
Caregiver Burden and Value Assessments in Duchenne Muscular Dystrophy
April 3rd 2025Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular dystrophy treatments was the focus of a recent International Society for Pharmacoeconomics and Outcomes Research (ISPOR) webinar.
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Sarepta Announces Positive Results for Elevidys, Gene Therapy for Duchenne
February 12th 2025Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is priced at $3.2 million per dose, produced $821 million in net product revenue for the company in 2024.
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