MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study
- Status
- UNKNOWN
- Phase
- N/A
Clinical Trials Related to Rare Diseases
Related studies linked through entity extraction aliases.
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
- Status
- COMPLETED
- Phase
- N/A
Study of A166 in Patients With Relapsed/Refractory Cancers Expressing HER2 Antigen or Having Amplified HER2 Gene
- Status
- COMPLETED
- Phase
- PHASE1/PHASE2
Dual Guidance Structure for Evaluation of Patients With Unclear Diagnosis in Centers for Rare Diseases
- Status
- COMPLETED
- Phase
- NA
A Trial For Participants With Ewing's Sarcoma Treated With Vigil in Combination With Irinotecan and Temozolomide
- Status
- TERMINATED
- Phase
- PHASE3
Diagnostic Research in Patients With Rare Diseases - Solving the Unsolved Rare Diseases
- Status
- UNKNOWN
- Phase
- N/A
Whole Genome Sequencing in the Detection of Rare Undiagnosed Genetic Diseases in Children in China
- Status
- UNKNOWN
- Phase
- N/A
The VetSeq Study: a Pilot Study of Genome Sequencing in Veteran Care
- Status
- COMPLETED
- Phase
- NA
Evaluation of HEArt invoLvement in Patients With FABRY Disease
- Status
- RECRUITING
- Phase
- N/A
Kidney Information Network for Disease Research and Education
- Status
- TERMINATED
- Phase
- N/A
NICUSeq: A Trial to Evaluate the Clinical Utility of Human Whole Genome Sequencing (WGS) Compared to Standard of Care in Acute Care Neonates and Infants
- Status
- COMPLETED
- Phase
- NA
Evaluate and Understand Preferences and Representations in Families of Patients With Regard to High-throughput Sequencing Technology for Diagnostic Purposes
- Status
- COMPLETED
- Phase
- NA
Pbi-shRNA™ EWS/FLI1 Type 1 LPX in Subjects With Advanced Ewing's Sarcoma
- Status
- COMPLETED
- Phase
- PHASE1
Finding Genes for Rare Diseases
- Status
- WITHDRAWN
- Phase
- N/A