Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

Summary

The intent of this protocol is to provide continued access to vamorolone for subjects in the United States and Canada who have completed the VBP15-LTE, VBP15- 004, or VBP15-006 protocols (and are thereby ineligible to enroll in another trial of vamorolone therapy), during the time a new drug application for vamorolone is under preparation and review.

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Publications

• Mah JK, Gonorazky HD, Nigro E, Lochmuller H, Aleman A, Yaworski A, Oskoui M, Sbrocchi AM, Selby KA, de Vera A, Mathur R, Gresko E, Linden A, Dutreix C, Hoffman EP. Vamorolone Safety, Pharmacokinetics, and Exploratory Efficacy in Duchenne Muscular Dystrophy: A Phase II, Nonrandomized, Multiple-Dose Study in 2-<4-Year-Old Boys. Neurology. 2026 Jun 9;106(11):e218066. doi: 10.1212/WNL.0000000000218066. Epub 2026 May 15.
• Lochmuller H, Gonorazky H, Nigro E, Mah JK, Aleman A, Yaworski A, Oskoui M, Sbrocchi AM, Selby K, de Vera A, McAdam L, Gresko E, Linden A, Dutreix C, Hoffman EP. Results of a phase II open-label, multiple-dose study of vamorolone (VBP15-006) in 7- to < 18-year-old boys with duchenne muscular dystrophy. J Neurol. 2026 Mar 3;273(3):177. doi: 10.1007/s00415-026-13711-6.

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