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| Name | Class |
|---|---|
| Clinigen Healthcare Limited | UNKNOWN |
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.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| delpacibart zotadirsen | Drug | Del-zota is administered as an IV infusion every 6 weeks. Doses are administered based on body weight. |
|
Key Inclusion Criteria
Rollover Participants
New (Non-Rollover) Participants
Key Exclusion Criteria
Rollover Participants
• Prescence of any new condition or worsening of existing condition that could affect participant's safety or ability to comply with the program requirements
New (Non-Rollover) Participants
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Clinigen Customer Service | Contact | 1 877 768 4303 | usmapcoordinators@clinigengroup.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama | Available | Birmingham | Alabama | 35294 | United States | |
| Arkansas Children's Hospital |
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| Available |
| Little Rock |
| Arkansas |
| 72202 |
| United States |
| Rare Disease Research | Available | Kissimmee | Florida | 34746 | United States |
| Nicklaus Children's Hospital | Available | Miami | Florida | 33155 | United States |
| Rare Disease Research | Available | Atlanta | Georgia | 30329 | United States |
| University of Kansas Medical Center | Available | Kansas City | Kansas | 66103 | United States |
| UMass Memorial Health Care | Available | Worcester | Massachusetts | 01583 | United States |
| Gillette Children's | Available | Saint Paul | Minnesota | 55101 | United States |
| Washington University | Available | St Louis | Missouri | 63110 | United States |
| Rare Disease Research | Available | Iselin | New Jersey | 08830 | United States |
| Nationwide Children's Hospital | Available | Columbus | Ohio | 43205 | United States |
| University of Pennsylvania | Available | Philadelphia | Pennsylvania | 19104 | United States |
| Prisma Health-Midlands Children's Hospital | Available | Columbia | South Carolina | 29203 | United States |
| University of Vermont Medical Center | Available | Burlington | Vermont | 05401 | United States |
| ID | Term |
|---|---|
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D040181 | Genetic Diseases, X-Linked |
| D009136 | Muscular Dystrophies |
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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