A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
- Status
- NOT_YET_RECRUITING
- Phase
- PHASE3
Clinical Trials Related to Genetic Diseases
Related studies linked through entity extraction aliases.
BAG3-DCM Natural History Study
- Status
- NOT_YET_RECRUITING
- Phase
- N/A
Impaired Type I IFN Immunity Due to Autoantibodies or a Genetic Defect: a Prospective National Cohort
- Status
- RECRUITING
- Phase
- N/A
PKP2-ACM Natural History Study
- Status
- RECRUITING
- Phase
- N/A
Decoding Developmental Disorders in Humams
- Status
- COMPLETED
- Phase
- N/A
Genotype-phenotype Characterization Study on Genetic Diseases With Immune and Neurological Dysfunctions
- Status
- RECRUITING
- Phase
- N/A
The Pilot Study of High-throughput Sequencing in Neonatal Birth Defects
- Status
- COMPLETED
- Phase
- N/A
Rapid Whole Genome Sequencing Study
- Status
- ENROLLING_BY_INVITATION
- Phase
- NA
Perinatal Precision Medicine
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- NA
Rady Children's Institute Genomic Biorepository
- Status
- RECRUITING
- Phase
- NA
Safety, Tolerability, Pharmacokinetics of ELX-02 in Healthy Adult Volunteers
- Status
- TERMINATED
- Phase
- PHASE1
The Alfred Step Test Exercise Protocol (A-STEP), for Adults With Cystic Fibrosis.
- Status
- COMPLETED
- Phase
- NA
Time-Lapse Incubation for Embryo Culture - Morphokinetics and Environmental Stability
- Status
- UNKNOWN
- Phase
- NA
Genetic and Metabolic Disease in Children
- Status
- RECRUITING
- Phase
- N/A
Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome
- Status
- COMPLETED
- Phase
- PHASE4
Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells
- Status
- TERMINATED
- Phase
- PHASE2
Mutation Exploration in Non-acquired, Genetic Disorders and Its Impact on Health Economy and Life Quality
- Status
- COMPLETED
- Phase
- N/A
Prenatal Microarray Follow-Up Study
- Status
- COMPLETED
- Phase
- N/A
Prenatal Cytogenetic Diagnosis by Array-Based Copy Number Analysis
- Status
- COMPLETED
- Phase
- N/A
Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics
- Status
- RECRUITING
- Phase
- N/A