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Results for “Deficiency Disease, Aspartoacylase”

6 trials

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Showing 6 of 6 results

Early research (Phase 1)UnknownNCT00278707
What this trial is testing

GTA-Glyceryltriacetate for Canavan Disease

Who this might be right for
Infantile Canavan DiseaseDeficiency Disease, Aspartoacylase
Sheba Medical Center 5
Testing effectiveness (Phase 2)WithdrawnNCT00657748
What this trial is testing

Lithium and Acetate for Canavan Disease

Who this might be right for
Canavan DiseaseInfantileDeficiency Disease+2 more
Assistance Publique - Hôpitaux de Paris
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
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
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Large-scale testing (Phase 3)Ended earlyNCT00654433
What this trial is testing

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

Who this might be right for
Inherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more
Aldagen 40