Abstract
With gene replacement therapy for Duchenne muscular dystrophy (DMD) being evaluated in phase 3 clinical trials, a crucial step is made to address numerous unmet needs of these patients. Since this therapy is associated with uncertainties regarding the benefits, side effects, and risks, this research wants to quantify the patient treatment needs and their acceptability towards treatment alternatives. The final objective is to inform regulatory, HTA/payer (reimbursement), and clinical practice decision making.
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Schedule a callMore From: Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
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