Rare diseases
Investigational molecules
Locations
Study on the Safety and Tolerability of Leriglitazone for Children with Rett Syndrome
SpainStudy of tovorafenib versus standard chemotherapy as first treatment for children with low-grade glioma with RAF gene changes
SpainStudy on Eteplirsen for Patients with Duchenne Muscular Dystrophy with Deletion Mutations Amenable to Exon 51 Skipping
SpainStudy of Setmelanotide for Patients with Obesity Due to Specific Genetic Variants
SpainStudy on Mavorixafor for Treating WHIM Syndrome in Patients
SpainStudy on the Effects of Spironolactone, Pioglitazone, and Metformin for Adolescent Girls and Young Women with Polycystic Ovary Syndrome (PCOS)
SpainStudy on the Effects of Ozanimod for Children with Moderate to Severe Crohn's Disease Not Responding to Usual Treatments
SpainStudy on the Long-Term Safety of PTC923 (Sepiapterin) for Patients with Phenylketonuria
SpainStudy on the Safety and Effectiveness of Ocrelizumab vs. Fingolimod for Children and Adolescents with Relapsing-Remitting Multiple Sclerosis
SpainStudy on Long-Term Spesolimab Treatment for Patients with Generalized Pustular Psoriasis from Previous Trials
SpainStudy on the Safety and Effectiveness of Baricitinib and Tocilizumab for Children with Systemic Juvenile Idiopathic Arthritis
SpainStudy on the Effectiveness and Safety of Crovalimab for Children with Atypical Hemolytic Uremic Syndrome (aHUS)
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