Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy

Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy

Abstract Introduction Duchenne muscular dystrophy (DMD) is a rare, progressive, debilitating neuromuscular disease. The early childhood onset and debilitating nature of the disease necessitate decades of caretaking for most patients. Caregivers have a critical role in evaluating patients’ physical f...

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Bibliographic Details
Main Authors: Craig M. McDonald, Jacob S. Elkins, Sai Dharmarajan, Katherine Gooch, Teofil Ciobanu, Claire J. Lansdall, Alexander P. Murphy, Fiona McDougall, Eugenio M. Mercuri, Ivana Audhya, the EMBARK Study Group
Format: Article
Language:English
Published: Adis, Springer Healthcare 2024-11-01
Series:Neurology and Therapy
Subjects:
Caregiver
Delandistrogene moxeparvovec
Duchenne muscular dystrophy
Gene therapy
Global Impressions scale
Online Access:https://doi.org/10.1007/s40120-024-00685-8
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