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Results for “TUBB4A-Related Leukodystrophy”

4 trials

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Showing 4 of 4 results

Testing effectiveness (Phase 2)Active Not RecruitingNCT07222371
What this trial is testing

An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy

Who this might be right for
Genetic DiseaseTUBB4A-Related Leukodystrophy
University of California, San Diego 1
Not applicableStudy completedNCT02699190
What this trial is testing

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Who this might be right for
LeukodystrophyWhite Matter Disease4H Syndrome+61 more
Children's Hospital of Philadelphia 236
Testing effectiveness (Phase 2)Not Yet RecruitingNCT06369974
What this trial is testing

Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

Who this might be right for
Genetic Disease
Massachusetts General Hospital 1
Not applicableLooking for participantsNCT03047369
What this trial is testing

The Myelin Disorders Biorepository Project

Who this might be right for
LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more
Children's Hospital of Philadelphia 12,000