| PHASE1 | 34 |
| PHASE1, PHASE2 | 12 |
| PHASE2 | 46 |
| PHASE2, PHASE3 | 3 |
| PHASE3 | 12 |
| PHASE4 | 8 |
| NA | 111 |
| Not Applicable | 144 |
| RECRUITING | 84 |
| ACTIVE_NOT_RECRUITING | 24 |
| NOT_YET_RECRUITING | 16 |
| COMPLETED | 172 |
| Databases | OMIMOrphanetClinicalTrialsPubMed |
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