25 Recruiting Genetic Disease Clinical Trials
Explore open interventional and observational research studies actively enrolling participants across the USA. Verify eligibility and contact site coordinators directly.
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Top Recruiting Genetic Disease Studies
Clinical Decision Support to Identify Pediatric Patients With Undiagnosed Genetic Disease
📍 Vanderbilt University Medical — Nashville, Tennessee
View Study & Checklist ➔Diagnostic Odyssey: Whole Genome Sequencing (WGS)
📍 Nickalus Children's Hospital f/k/a Miami Children's Hospital — Miami, Florida
View Study & Checklist ➔The Electronic Medical Records and GEnomics (eMERGE) Network Genomic Risk Assessment
📍 University of Alabama Birmingham — Birmingham, Alabama
View Study & Checklist ➔Penn Medicine Biobank Return of Results Program
📍 University of Pennsylvania — Philadelphia, Pennsylvania
View Study & Checklist ➔An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy
📍 Rady Children's Hospital San Diego — San Diego, California
View Study & Checklist ➔Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A
📍 Rady Children's Hospital — San Diego, California
View Study & Checklist ➔Adaptive Optics Retinal Imaging in Inherited and Acquired Retinal Disorders
📍 The Hospital for Sick Children — Toronto, Ontario
View Study & Checklist ➔Delineating the Molecular Spectrum and the Clinical, Imaging and Neuronal Phenotype of Chopra-Amiel-Gordon Syndrome
📍 Boston Children's Hospital — Boston, Massachusetts
View Study & Checklist ➔STXBP1 and SYNGAP1 Related Disorders Natural History Study
📍 Stanford Medicine Children's Health — Palo Alto, California
View Study & Checklist ➔An Adaptive Clinical Trial of BeginNGS Newborn Screening for Hundreds of Genetic Diseases by Genome Sequencing
📍 Rady Children's Hospital San Diego — San Diego, California
View Study & Checklist ➔Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network
📍 University of Alabama at Birmingham — Birmingham, Alabama
View Study & Checklist ➔