Caregiver-reported Patient Experiences with Duchenne Muscular Dystrophy: Qualitative In-trial Interviews 1 Year After Delandistrogene Moxeparvovec in the Pivotal EMBARK Trial

Abstract Introduction Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disease. The first approved DMD gene therapy, delandistrogene moxeparvovec, was evaluated in the phase 3, two-part, randomized, placebo-controlled EMBARK trial (NCT05096221), which did not meet statistical sign...

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Үндсэн зохиолчид:	Ivana Audhya, Alise B. Nacson, Katherine Gooch, Bonita Basnyat, Christina Slota, Susan Martin, Alex Murphy, Claire J. Lansdall, Teofil Ciobanu, Andres Nascimento, Aravindhan Veerapandiyan
Формат:	Artigo
Хэл сонгох:	Inglês
Хэвлэсэн:	Adis, Springer Healthcare 2025-11-01
Цуврал:	Neurology and Therapy
Нөхцлүүд:	Caregiver Global Impression of Change (CaGI-C) measure Caregiver Global Impression of Severity (CaGI-S) measure Caregivers Duchenne muscular dystrophy EMBARK Global impression
Онлайн хандалт:	https://doi.org/10.1007/s40120-025-00842-7
Шошгууд:	Шошго нэмэх Шошго байхгүй, Энэхүү баримтыг шошголох эхний хүн болох!