The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.
Completed Study EXPLORE44-OLE Treatment Period (through W102)
No significant tolerability issues with AOC 1044
Permanently residing in the US and have a US primary health care provider
Documented dystrophin gene mutation that is amenable to exon 44 skipping
Recently treated with or on a clinical study for another investigation drug
Serious respiratory or cardiac dysfunction, or nearing end of life
Screening laboratory parameters do not meet protocol requirements
History of multiple drug allergies or to any component of AOC 1044
Avidity Biosciences, Inc.
Lead sponsor
Clinigen Healthcare Limited
Collaborator