The goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD).
Patient or parent/legal guardian is willing and able to provide written informed consent once the nature of the registry has been explained and prior to the start of any registry-related procedures.
Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
Male patients at least 2 years old.
Confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to anti- dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
None
Catalyst Pharmaceuticals, Inc.
Lead sponsor
ICON plc
Collaborator
Veeva Systems
Collaborator
Children's Hospital of Eastern Ontario
Collaborator
Medpace, Inc.
Collaborator