An expanded access record providing delpacibart zotadirsen in Muscular Disorders, Atrophic, Muscular Diseases and Musculoskeletal Diseases, sponsored by Avidity Biosciences, Inc.. Available at 22 sites in United States. Open to male participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2026-10-08.
Sponsored by Avidity Biosciences, Inc. (part of Novartis) · Expanded access
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.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 108 are open to participants now.
Browse Muscular Dystrophy, Duchenne studies →Avidity Biosciences, Inc. is the lead sponsor of 11 studies on the registry; 4 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 1 (20%) have results posted.
First submitted before Avidity Biosciences, Inc. became part of Novartis.
Compared within Muscular Dystrophy, Duchenne, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Muscular Disorders, Atrophic. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Key Inclusion Criteria
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Key Exclusion Criteria
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New (Non-Rollover) Participants
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Del-zota is administered as an IV infusion every 6 weeks. Doses are administered based on body weight.
Also known as: Del-zota, AOC 1044
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Muscular Dystrophy, Duchenne →