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A Phase 2 Open-label, Dose Escalation Study of HST5040 in Subjects with Propionic or Methylmalonic Acidemia Followed by a 6-Month, Randomized, Double-blind, Placebo-controlled, 2-period Crossover Study and an Open-label, Long-term Extension Study Drug Clinical Trial

Randomized, Phase 3, double-blind, 52-week study to evaluate the efficacy and safety of rilzabrutinib (SAR444671) compared to placebo in adult participants with active IgG4- related disease Drug Clinical Trial

A 3 Month, Multicenter, Double-Masked Safety and Efficacy Study of Travoprost Ophthalmic Solution, 0.004% Compared to Timolol (0.5% or 0.25%) in Pediatric Glaucoma Patients Drug Clinical Trial

A 3 Month, Multicenter, Double-Masked Safety and Efficacy Study of Travoprost Ophthalmic Solution, 0.004% Compared to Timolol (0.5% or 0.25%) in Pediatric Glaucoma Patients Drug Clinical Trial

A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to < 18 years of age, treatment naive, sitting, and never ambulatory Drug Clinical Trial

A 52-week, double-blind, randomised, multi-centre, phase III, parallel-group study in patients 12 years and older with asthma, evaluating the efficacy and safety of Symbicort (budesonide/formoterol) Turbuhaler 160/4.5 μg 'as needed' compared with Pulmicort (budesonide) Turbuhaler 200 μg twice daily plus terbutaline Turbuhaler 0.4 mg 'as needed' Drug Clinical Trial

A randomized, double-blind, placebo-controlled phase II study to investigate the efficacy and safety of riociguat (0.5 mg, 1.0 mg, 1.5 mg, 2.0 mg and 2.5 mg TID) in patients with symptomatic pulmonary hypertension associated with idiopathic interstitial pneumonias (IIP). Drug Clinical Trial

A 52-week, double-blind, randomised, multi-centre, phase III, parallel-group study in patients 12 years and older with asthma, evaluating the efficacy and safety of Symbicort (budesonide/formoterol) Turbuhaler 160/4.5 μg 'as needed' compared with Pulmicort (budesonide) Turbuhaler 200 μg twice daily plus terbutaline Turbuhaler 0.4 mg 'as needed' Drug Clinical Trial

A randomized, double-blind, placebo-controlled phase II study to investigate the efficacy and safety of riociguat (0.5 mg, 1.0 mg, 1.5 mg, 2.0 mg and 2.5 mg TID) in patients with symptomatic pulmonary hypertension associated with idiopathic interstitial pneumonias (IIP). Drug Clinical Trial

Multicenter, open-label, study to evaluate safety,tolerability, pharmacokinetics and, pharmacodynamics of LCZ696 followed by a 52-week randomized, double-blind, parallel group, active-controlled study to evaluate the efficacy and safety of LCZ696 compared with enalapril in pediatric patients from1 month to < 18 years of age with heart failure due to systemic left ventricle systolic dysfunction) Drug Clinical Trial