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"A Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched and HLA-Matched Bone Marrow for Patients with Sickle Cell Disease and Other Hemoglobinopathies " Drug Clinical Trial

A PARTIALLY DOUBLE-BLINDED, RANDOMIZED, CONTROLLED, PLACEBO-CONTROLLED STUDY, A PHASE III TRIAL OF SAFETY AND EFFICACY OF PREMEDICATION WITH DEXMEDETOMIDINE AND MIDAZOLAM IN PEDIATRIC PATIENTS Drug Clinical Trial

A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations Drug Clinical Trial

A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care Drug Clinical Trial

"A Phase 2/3, Randomized, Double-blind, Placebo- and Active-controlled, Parallel-group, Multicenter Protocol to Evaluate the Efficacy and Safety of Guselkumab in Participants with Moderately to Severely Active Crohn's Disease " Drug Clinical Trial

A PHASE 2A, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED, PARALLEL GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ORAL PF-06651600 AND PF 06700841 AS INDUCTION AND OPEN LABEL EXTENSION TREATMENT IN SUBJECTS WITH MODERATE TO SEVERE CROHN’S DISEASE Drug Clinical Trial

A Phase 3, Double-Blind, Randomized, Efficacy and Safety Comparison of Prasugrel and Placebo in Pediatric Patients With Sickle Cell Disease. Drug Clinical Trial

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study To Determine The Efficacy And Safety Of Luspatercept (Ace-536) Versus Placebo In Adults Who Require Regular Red Blood Cell Transfusions Due To Beta -Thalassemia The “BELIEVE” Trial Drug Clinical Trial

"A Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled Clinical Study to Evaluate the Safety and Efficacy of Ertugliflozin (MK-8835/PF-04971729) in Pediatric Participants (ages 10 to 17 years, inclusive) with Type 2 Diabetes Mellitus" Drug Clinical Trial

A Phase 3, Multicenter, Open-Label Extension Study of Oral Ozanimod for Moderately to Severely Active Crohn’s Disease Drug Clinical Trial