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Results for “BPAN”

7 trials

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Enrolling by Invitation

Showing 7 of 7 results

Not applicableLooking for participantsNCT05522374
What this trial is testing

TIRCON International NBIA Registry

Who this might be right for
Neurodegeneration With Brain Iron Accumulation (NBIA)Pantothenate Kinase-associated Neurodegeneration (PKAN)Beta-Propeller Protein-Associated Neurodegeneration (BPAN)+8 more
LMU Klinikum 2,000
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 applicableUnknownNCT03572114
What this trial is testing

Imaging Neuromelanin and Iron in Dystonia/Parkinsonism

Who this might be right for
Sporadic DystoniaDystonia, FamilialParkinson Disease, Juvenile+2 more
University College, London 80
Not applicableStudy completedNCT05954494
What this trial is testing

Protection Response to Cellular Stress in BPAN's Patient Study

Who this might be right for
Children and Adults With BPAN Carrying Pathogenic Variants of WDR45
Hospices Civils de Lyon 21
Not applicableUnknownNCT02587858
What this trial is testing

NBIAready: Online Collection of Natural History Patient-reported Outcome Measures

Who this might be right for
Neurodegeneration With Brain Iron Accumulation (NBIA)
Susan J. Hayflick 300
Not applicableEnrolling By InvitationNCT06938542
What this trial is testing

Palliative Care Needs of Children With Rare Diseases and Their Families

Who this might be right for
Trisomy 13 SyndromeArthrogryposis Congenita Multiplex With Intestinal AtresiaAsparagine Synthetase Deficiency+16 more
Children's National Research Institute 480
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