A Randomized, Open-label, Multicentral, Two Arm, Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet) Read more about A Randomized, Open-label, Multicentral, Two Arm, Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)
Phase 2, Single-Site, Open-Label, Randomized, 2-ArmParallel Study to Assess the Efficacy and Safety ofRyanodex® (EGL-4104) as Adjuvant Treatment inSubjects With Exertional Heat Stroke (EHS Read more about Phase 2, Single-Site, Open-Label, Randomized, 2-ArmParallel Study to Assess the Efficacy and Safety ofRyanodex® (EGL-4104) as Adjuvant Treatment inSubjects With Exertional Heat Stroke (EHS
An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 (Sebelipase Alfa) in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency Read more about An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 (Sebelipase Alfa) in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy and Pharmacokinetics of Asfotase Alfa (human recombinant tissue non-specific alkaline phosphatase fusion protein) in Infants and Children ≤ 5 Years of Age with Hypophosphatasia (HPP). Read more about An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy and Pharmacokinetics of Asfotase Alfa (human recombinant tissue non-specific alkaline phosphatase fusion protein) in Infants and Children ≤ 5 Years of Age with Hypophosphatasia (HPP).
