Duplication Clinical Trials in Philadelphia, Pennsylvania
4 recruitingPhiladelphia, Pennsylvania
Showing 1–4 of 4 trials
Recruiting
Phase 1Phase 2
ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Ionis Pharmaceuticals, Inc.48 enrolled11 locationsNCT06430385
Recruiting
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
Mitochondrial DiseasesMitochondrial DisordersMitochondrial Genetic Disorders+2 more
Columbia University1,000 enrolled17 locationsNCT01694940
Recruiting
The Myelin Disorders Biorepository Project
AdrenoleukodystrophyCockayne SyndromeRefsum Disease+63 more
Children's Hospital of Philadelphia12,000 enrolled23 locationsNCT03047369
Recruiting
Characterization and Natural History of Williams Syndrome and Other Chromosome 7q11.23 Variants
Williams SyndromeWilliams Beuren SyndromeWilliams Beuren Region Duplication+1 more
University of Pennsylvania2,000 enrolled1 locationNCT06930417