Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy.

Description

This is a multi-center, observational, prospective, longitudinal registry designed to collect data in male patients aged 2 years and older with DMD treated with AGAMREE®.

This registry will be conducted in the US, at approximately 25 sites known to treat and follow patients with DMD. The registry plans to enroll approximately 250 male patients aged 2 years and older with DMD.

Evaluations will include:

* Growth parameters
* Body mass index (BMI)
* Vital Signs
* Physical Exam
* Laboratory (Chemistry and Hematology)
* North Star Ambulatory Assessment (NSAA)
* Performance of Upper Limb (PUL)
* Cardiovascular status
* Fractures
* Bone density
* Puberty
* Quality of life (QoL)
* Adverse events (AEs)

Patients will be followed for approximately 5 years in the registry and will return to the site for Yearly Follow-up Visits (+/- 30 days) for registry assessments. Information on standard of care treatment and procedures for management of DMD will also be collected. Patients and/or their parents/legal guardians will be asked to complete paper QoL questionnaires at enrollment and at each Yearly Follow-up Visit (+/- 30 days).