This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
13
Single IV infusion of delandistrogene moxeparvovec
Chr de La Citadelle
Liège, Belgium
Hôpital Necker-Enfants Malades
Paris, France
Universitätsklinikum Essen
Essen, Germany
PU A. Gemelli, Università Cattolica del Sacro Cuore
Rome, Lazio, Italy
Percentage of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
Time frame: Baseline up to Week 260
Change in Quantity of Delandistrogene Moxeparvovec Dystrophin as Measured by Western Blot
Time frame: Baseline, Week 12
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Hospital Sant Joan De Deu
Esplugues de Llobregas, Barcelona, Spain
Great Ormond Street Hospital for Children
London, United Kingdom
John Radcliffe Hospital
Oxford, United Kingdom