Rare diseases
Investigational molecules
Locations
Efficacy and Safety of Givinostat for Non-Ambulant Patients with Duchenne Muscular Dystrophy
SpainStudy on the Safety and Effectiveness of Quizartinib for Children with Newly Diagnosed FLT3-ITD Positive AML and Normal NPM1
SpainStudy on the Safety and Effects of Radiprodil for Children with GRIN-related Disorder
SpainStudy on Alpelisib for Treating Lymphatic Malformations in Patients with PIK3CA Mutation
SpainStudy on Crohn's Disease: Evaluating Risankizumab's Effects in Children Aged 2 to 17 with Moderate to Severe Symptoms
SpainStudy on Gene Therapy with AAV9.LAMP2B for Male Patients with Danon Disease
SpainStudy on Risankizumab for Children Aged 6-17 With Moderate to Severe Plaque Psoriasis After Completing Previous Study
SpainStudy of Aztreonam-Avibactam and Metronidazole for Children with Serious Gram-Negative Bacterial Infections
SpainStudy on Imlifidase and Drug Combination for Kidney Transplant in Highly Sensitized Children
SpainStudy on Vericiguat for Children with Heart Failure Due to Left Ventricular Systolic Dysfunction
SpainStudy of ONC201 for Patients with Newly Diagnosed H3 K27M-mutant Diffuse Glioma After Radiotherapy
SpainStudy of Lutetium (177Lu) Oxodotreotide for Children with Relapsed or Refractory High-Risk Neuroblastoma
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