Rare diseases
Investigational molecules
Locations
Study of Apitegromab Treatment in Children Under 2 Years Old with Spinal Muscular Atrophy (SMA)
SpainStudy of alpelisib in children and adults with PIK3CA-Related Overgrowth Spectrum (PROS)
SpainLong-term safety and effectiveness study of ritlecitinib in children with severe alopecia areata who completed previous trials B7981027 or B7981031
SpainStudy of intrathecal S230815 to evaluate safety and effectiveness in children with KCNT1-related Developmental and Epileptic Encephalopathy
SpainStudy of belumosudil in children aged 1 to 18 years with moderate to severe chronic graft versus host disease requiring systemic treatment
SpainStudy of drug combination therapy in children and adolescents with relapsed or refractory B-cell non-Hodgkin lymphoma using odronextamab, loncastuximab tesirine and rituximab
SpainStudy of efgartigimod given by IV infusion in adolescents aged 12-17 years with chronic immune thrombocytopenia
SpainStudy of NVD003 stem cell implant compared to bone graft for children with congenital pseudarthrosis of the tibia
SpainEvaluating Different Durations of Antibiotic Treatment for Respiratory Tract Infections and Rhinosinusitis in Adults Receiving Beta-lactam Antibiotics
SpainStudy of Atacicept Treatment for Patients with Multiple Autoimmune Glomerular Diseases
SpainEvaluation of Enoxaparin for Prevention of Localized Intravascular Coagulopathy in Children with Slow-Flow Vascular Malformations Undergoing Interventional Procedures
SpainStudy of Etranacogene Dezaparvovec (CSL222) in Adolescent Males with Severe or Moderately Severe Hemophilia B
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