A Multi-Dimensional Model of cAre and transItion for Patients With cOmplex RAre Diseases
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- N/A
Clinical Trials Related to Rare Diseases
Related studies linked through entity extraction aliases.
Evaluation of Socio-professional Inclusion for Young Adults Aged 15-25 Living With a Rare Genetic Disability
- Status
- RECRUITING
- Phase
- N/A
A Longitudinal Study on Family Adaptation and Relationship Dynamics in Pediatric Rare Diseases
- Status
- NOT_YET_RECRUITING
- Phase
- N/A
Implementation of Long-read Sequencing for the Diagnosis of Rare Diseases.
- Status
- NOT_YET_RECRUITING
- Phase
- N/A
Epidemiological Study of Treatment Approaches on AQP4-IgG Positive NMOSD in Russia
- Status
- SUSPENDED
- Phase
- N/A
Epidemiological Study of Treatment Approaches in AChR-Antibody Positive Generalized Myasthenia Gravis in Russia
- Status
- RECRUITING
- Phase
- N/A
Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center
- Status
- RECRUITING
- Phase
- N/A
Genetic Study to Determine the Cause of Birth Defects in Newborns in Texas
- Status
- RECRUITING
- Phase
- NA
Identification of Cellular Biomarkers of Rare Eye Diseases in Adults
- Status
- NOT_YET_RECRUITING
- Phase
- NA
Genomic Profiling of Genetic and Rare Diseases
- Status
- RECRUITING
- Phase
- NA
Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation
- Status
- RECRUITING
- Phase
- EARLY_PHASE1
BEhavioral and Adherence Model for Improving Quality, Health Outcomes and Cost-Effectiveness of healthcaRe
- Status
- RECRUITING
- Phase
- NA
PREcision Diagnostics in Rare genetIC Diseases and Tumors - Long Read Sequencing
- Status
- RECRUITING
- Phase
- N/A
Fibroblasts and Thoracic Aortic Aneurysms: in Vitro Characterization in With Marfan Syndrome and Genetic Aortic Diseases
- Status
- ENROLLING_BY_INVITATION
- Phase
- N/A
Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)
- Status
- RECRUITING
- Phase
- PHASE2
Evaluation of the French Version of the PEERS Social Skills Training Program for Adolescents With Rare Neurodevelopmental Disorders
- Status
- COMPLETED
- Phase
- N/A
Genetic Analysis of Uncommon Disease Presentations in Non-US Populations
- Status
- RECRUITING
- Phase
- N/A
Institutional Registry of Rare Diseases
- Status
- RECRUITING
- Phase
- N/A
Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia
- Status
- WITHDRAWN
- Phase
- N/A
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
- Status
- RECRUITING
- Phase
- N/A