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Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network
- University of California, Los Angeles, Los Angeles
- University of California, Irvine Medical Center, Orange
- Stanford University, Stanford
- ak 2 plis
Ap rekriteEtid obsèvasyonVolontè an sante akeyi
1 Month – 100 Years · Tout sèks · Mizajou Oct 2, 2026 · NCT02450851Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
- UCSF Benioff Children's Hospital Oakland, Oakland
Ap rekriteFaz 2/3
Laj 2 a 17 · Tout sèks · Mizajou Sep 24, 2026 · NCT07441876A Long-Term Observational Study of Patients With Fucosidosis
- Children's Hospital of Orange County, Orange
Ap rekriteEtid obsèvasyon
Tout laj · Tout sèks · Mizajou Sep 17, 2026 · NCT07615400Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
- UCLA Ataxia Center, Los Angeles
Ap rekriteEtid obsèvasyonVolontè an sante akeyi
Tout laj · Tout sèks · Mizajou Sep 1, 2026 · NCT06016946A Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD)
- GC2501 Study Site 100, Los Angelas
Ap rekriteFaz 3
Laj 18 a 80 · Tout sèks · Mizajou Aug 11, 2026 · NCT07555483Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
- University of California Irvine, Orange
Ap rekriteFaz 2
Laj 18 ak plis · Tout sèks · Mizajou Aug 5, 2026 · NCT07224360Prospective Clinical Assessment Study in Children With Hypochondroplasia
- UCSF Benioff Children's Hospital, Oakland
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30 Months – 16 Years · Tout sèks · Mizajou Jul 16, 2026 · NCT06410976Rett Syndrome Registry
- Children's Hospital Los Angeles, Los Angeles
- UCSF Benioff Children's Hospital, Oakland
- Rady Children's Hospital - San Diego, San Diego
Ap rekriteEtid obsèvasyon
Laj 0 a 99 · Tout sèks · Mizajou Jun 30, 2026 · NCT05432349A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
- Ronald Reagan UCLA Medical Center, Los Angeles
- University of Stanford Medical Center, Palo Alto
Ap rekriteFaz 1/2
Laj 1 ak plis · Tout sèks · Mizajou Mar 23, 2026 · NCT05130437Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
- UCSD Altman Clinical and Transalational Research Institute Building, Los Angeles
- Ronald Reagan UCLA Medical Center, Los Angeles
- Lucile Packard Children's Hospital Stanford, Stanford
Ap rekriteFaz 1/2
Tout laj · Tout sèks · Mizajou Jan 22, 2026 · NCT04159103An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705
- UCLA Medical Center, Los Angeles
- Lucile Packard Children's Hospital at Stanford, Palo Alto
- Altman Clinical and Translational Research Institution, San Diego
Ap rekriteFaz 1/2
Laj 1 ak plis · Tout sèks · Mizajou Dec 23, 2025 · NCT05295433STXBP1 and SYNGAP1 Related Disorders Natural History Study
- Stanford Medicine Children's Health, Palo Alto
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Tout laj · Tout sèks · Mizajou Oct 29, 2025 · NCT06555965Large Language Models To Improve the Quality of Care of Cardiology Patients
- Stanford, Palo Alto
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Laj 18 ak plis · Tout sèks · Mizajou May 15, 2025 · NCT06935253Natural History Study of Patients with HPDL Mutations
- Eun Hae Lee, San Diego
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Tout laj · Tout sèks · Mizajou Mar 30, 2025 · NCT05848271An Adaptive Clinical Trial of BeginNGS Newborn Screening for Hundreds of Genetic Diseases by Genome Sequencing
- Rady Children's Hospital San Diego, San Diego
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1 Day – 28 Days · Tout sèks · Mizajou Mar 12, 2024 · NCT06306521Rady Children's Institute Genomic Biorepository
- Rady Pediatric Genomics & Systems Medicine Institute, San Diego
Ap rekriteVolontè an sante akeyi
Tout laj · Tout sèks · Mizajou Dec 12, 2022 · NCT02917460Sous ClinicalTrials.gov, done yo ranmase Oct 5, 2026. Chak etid gen kritè li pou li menm; ekip etid la deside ki moun ki ka patisipe.