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

Study Summary

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).

Conditions

Duchenne Muscular Dystrophy

Interventions / Drugs

Vamorolone

View on ClinicalTrials.gov ↗

Sponsor

CPRX

Catalyst Pharmaceuticals

Rare Disease

F35/100

Study Details

Enrollment250 participants

Primary Completion—

Lead SponsorCatalyst Pharmaceuticals, Inc.

Data Retrieved2026-04-11 04:10:36.012836+00:00

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