Rare diseases
Investigational molecules
Locations
Study on the Safety and Efficacy of Viltolarsen for Boys with Duchenne Muscular Dystrophy
SpainStudy on Tisagenlecleucel for High-Risk B-Cell Acute Lymphoblastic Leukemia in Pediatric and Young Adult Patients with Minimal Residual Disease
SpainStudy of Abemaciclib, Irinotecan, and Temozolomide for Patients with Relapsed or Refractory Ewing's Sarcoma
SpainStudy on the Effectiveness and Safety of Obinutuzumab and Mycophenolate Mofetil in Children with Idiopathic Nephrotic Syndrome
SpainStudy on the Effects and Safety of INZ-701 for Children with ENPP1 Deficiency
SpainStudy on the Effects and Safety of Emicizumab for Infants with Hemophilia A (Ages Birth to 12 Months)
SpainStudy on the Safety and Effects of INZ-701 for Infants with ENPP1 or ABCC6 Deficiency
SpainStudy on Vericiguat for Children with Heart Failure from Left Ventricular Systolic Dysfunction
SpainStudy on the Safety and Immune Response of V116 Vaccine in Children and Adolescents at Higher Risk for Pneumococcal Disease
SpainStudy on Finerenone for Children with Chronic Kidney Disease and Proteinuria Using ACEI or ARB
SpainStudy on Lasmiditan for Treating Migraine in Children Aged 6 to 17
SpainStudy on Long-Term Safety and Effects of Apitegromab in Patients with Type 2 and TYPE 3 Spinal Muscular Atrophy Who Completed Previous Trials
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