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Single arm Phase I/II study of the safety, tolerability and efficacy of the investigational anti PD-L1 monoclonal antibody Durvalumab in combination with Paclitaxel in patients with metastatic triple negative PD-L1 positive breast cancer Drug Clinical Trial

A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) Drug Clinical Trial

A Phase III, Double-Blind, Randomised Study to Assess the Efficacy and Safety of AZD9291 versus a Standard of Care Epidermal Growth Factor Receptor Tyrosine Kinase Inhibitor as First-Line Treatment in Patients with Epidermal Growth Factor Receptor Mutation Positive, Locally Advanced or Metastatic Non-Small Cell Lung Cancer Drug Clinical Trial

Single arm Phase I/II study of the safety, tolerability and efficacy of the investigational anti PD-L1 monoclonal antibody Durvalumab in combination with Paclitaxel in patients with metastatic triple negative PD-L1 positive breast cancer Drug Clinical Trial

An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease Drug Clinical Trial

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Arm Study to Evaluate the Efficacy and Safety of RZ358 in Patients with Congenital Hyperinsulinism Drug Clinical Trial

A randomized, double-blind, Phase 3 study comparing efficacy and safety of frexalimab (SAR441344) to placebo in adult participants with nonrelapsing secondary progressive multiple sclerosis. Drug Clinical Trial

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Voxelotor (GBT440) in Pediatric Participants with Sickle Cell Disease (HOPE Kids 2) Drug Clinical Trial

A prospective, randomized, double-blind, multicenter, placebo-controlled, parallel group, adaptive Phase 3 study with open-label extension to evaluate efficacy and safety of macitentan 75 mg in inoperable or persistent/recurrent chronic thromboembolic pulmonary hypertension. Drug Clinical Trial

Phase II study of neoadjuvant hormonal therapy guided by interval Ki67 testing in hormonal receptor-positive, HER2-negative breast cancer. Drug Clinical Trial