| A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (NCT05126758) | Capricor Inc. | 2022-06-22 |
| Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) (NCT05356195) | Vertex Pharmaceuticals Incorporated | 2022-05-03 |
| Evaluating Prenatal Exome Sequencing Study (NCT05290701) | Leiden University Medical Center | 2022-02-21 |
| Genetic Diagnosis and Human Growth Hormone Treatment in Small for Gestational Age Children With Short Stature (NCT05070234) | Chunxiu Gong | 2021-10-11 |
| A Long-term Follow-up Study in Participants Who Received CTX001 (NCT04208529) | Vertex Pharmaceuticals Incorporated | 2021-01-20 |
| Guanfacine Extended Release for the Reduction of Aggression and Self-injurious Behavior Associated With Prader-Willi Syndrome (NCT05657860) | Maimonides Medical Center | 2020-12-17 |
| Whole Genome Sequencing in the Neonatal Intensive Care Unit (NCT03721458) | Milton S. Hershey Medical Center | 2019-05-28 |
| NYCKidSeq: Incorporating Genomics Into Clinical Care of Diverse NYC Children (NCT03738098) | Icahn School of Medicine at Mount Sinai | 2019-01-30 |
| Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors (NCT03734588) | Spark Therapeutics, Inc. | 2019-01-30 |
| Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants (NCT03876301) | Spark Therapeutics, Inc. | 2019-01-21 |
| Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia (NCT03718234) | University of Minnesota | 2019-01-01 |
| Study Evaluating Efficacy and Safety of PF-04965842 and Dupilumab in Adult Subjects With Moderate to Severe Atopic Dermatitis on Background Topical Therapy (NCT03720470) | Pfizer | 2018-10-29 |
| A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia (NCT03655678) | Vertex Pharmaceuticals Incorporated | 2018-09-14 |
| Effect of Benralizumab in Atopic Dermatitis (NCT03563066) | McMaster University | 2018-09-04 |
| Study to Investigate Efficacy and Safety of PF-04965842 in Subjects Aged 12 Years and Over With Moderate to Severe Atopic Dermatitis With the Option of Rescue Treatment in Flaring Subjects (NCT03627767) | Pfizer | 2018-06-11 |
| A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (NCT03406780) | Capricor Inc. | 2018-04-04 |
| Evaluating Innovations in Transition From Pediatric to Adult Care - The Transition Navigator Trial (NCT03342495) | University of Calgary | 2018-02-06 |
| Study on the Mechanism of Neurodevelopment Dysplasia of Fetal Brain Caused by ASNS Gene Mutation (NCT03587155) | The First Hospital of Jilin University | 2017-10-28 |
| Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy (NCT03179631) | PTC Therapeutics | 2017-07-06 |
| Uterine Irrigation Method in Infertility Patients Who Require PGD (NCT02676479) | Istanbul University | 2016-02-01 |
| Natural History Study of Children With Metachromatic Leukodystrophy (NCT01963650) | Shire | 2015-11-02 |
| Utilizing Free DNA in Embryo Culture for PGT (NCT02884063) | Al Baraka Fertility Hospital | 2015-05-01 |
| An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy (NCT02090959) | PTC Therapeutics | 2014-03-20 |
| Pilot Study of Cardiac MR in Patients With Muscular Dystrophy (NCT02921321) | Children's National Research Institute | 2014-01-01 |
| Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) (NCT01826487) | PTC Therapeutics | 2013-03-26 |
| An Analysis of the Symptomatic Domains Most Relevant to Charcot Marie Tooth Neuropathy (CMT) Patients (NCT02429947) | University of South Florida | 2012-07-01 |
| An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia (NCT00694109) | Kastle Therapeutics, LLC | 2008-04-01 |
| Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia (NCT00607373) | Kastle Therapeutics, LLC | 2007-07-01 |
| Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia (NCT00477594) | Kastle Therapeutics, LLC | 2007-05-01 |
| High-Density Lipoprotein (HDL) Treatment Study (NCT00458055) | McGill University Health Centre/Research Institute of the McGill University Health Centre | 2006-11-01 |
| Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration (NCT00362180) | Kastle Therapeutics, LLC | 2006-07-01 |
| Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS) (NCT00288704) | Regeneron Pharmaceuticals | 2005-12-01 |
| Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping (NCT07250737) | Avidity Biosciences, Inc. | |