Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

Vamorolone will be shipped to the subject's family by the study site. The patient will
receive standard of care treatment and procedures for management of DMD. Treating Physicians
participating in the expanded access program are required to collect and document any
physician, patient, or caregiver reported safety events and report to the Sponsor. The
subject's dose of vamorolone may be increased or decreased within a range of 2.0 to 6.0
mg/kg/day (only doses of 2 mg/kg, 4 mg/kg and 6 mg/kg are allowed), given once daily.
Administration of vamorolone (taken with an 8 ounce (240 ml) glass of full fat milk, or
equivalent high-fat food portion) will be unchanged from the VBP15-LTE or VBP15-004 studies.
In the absence of safety concerns, and while this Expaneded Access protocol is active,
vamorolone may be provided indefinitely or until approval, provided that the Treating
Physician and family agrees that continued administration of vamorolone is in the best
interest of the child

Inclusion Criteria:

- Subject's parent or legal guardian has provided written informed consent/HIPAA
authorization

- Subject has previously completed at a participating US study site VBP15-LTE up to and
including the Month 24 assessments, OR VBP15-004 up to and including the Week 48
assessments, within 30 days prior to participation in the VBP15-EAP

- Subject and parent/guardian are willing and able to comply with recommended study drug
administration plan, and standard of care follow-up and monitoring as recommended by
their Treating Physician

Exclusion Criteria:

- Subject had a serious or severe adverse event in study VBP15-LTE or VBP15-004 that, in
the opinion of the Treating Physician and Sponsor, was probably or definitely related
to vamorolone use and precludes safe use of vamorolone for the subject in this
expanded access program

- Subject and/or parent/guardian are unable and/or unwilling to comply with regular
medical care and follow-up as recommended by their Treating Physician throughout
participation in the VBP15-EAP