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Study of ORL-1M in Patients With CDG-Ib
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment with ORL-1M - D-mannose | Other |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ORL-1M - D-mannose | Drug | Oral ORL-1M |
|
| Measure | Description | Time Frame |
|---|---|---|
| Improvement in hypoglycemia, diarrhea and vomiting. | Decreased frequency of hypoglycemic episode, diarrhea and vomiting frequency. | 6 months after treatment started |
| Measure | Description | Time Frame |
|---|---|---|
| Improved glycosylation pattern of serum transferrin. | Normalized isoelectric focusing pattern of serum transferrin. | 30 days after treatment started |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Study Coordinator Study Coordinator | Contact | +90 537 763 6241 | business.development@orphalabs.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Orpha Labs | Recruiting | Ankara | 06100 | Turkey (Türkiye) |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35120836 | Derived | Rebelo AL, Chevalier MT, Russo L, Pandit A. Role and therapeutic implications of protein glycosylation in neuroinflammation. Trends Mol Med. 2022 Apr;28(4):270-289. doi: 10.1016/j.molmed.2022.01.004. Epub 2022 Feb 1. |
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| ID | Term |
|---|---|
| C535740 | Congenital disorder of glycosylation type 1B |
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