Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study of Risdiplam for Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
BelgiumStudy on the Safety and Effects of Setanaxib for Patients with Alport Syndrome
AustriaStudy on the Safety and Tolerance of BI 1291583 in Adults with Cystic Fibrosis Bronchiectasis
BelgiumStudy on Soticlestat for Children and Adults with Dravet or Lennox-Gastaut Syndrome Previously Treated with Fenfluramine
DenmarkStudy on Cannabidiol Oral Solution for Infants with Tuberous Sclerosis, Dravet Syndrome, or Lennox-Gastaut Syndrome Experiencing Uncontrolled Seizures
ItalyStudy on Larotrectinib for Treating Children with Advanced Solid Tumors with NTRK Fusion
CzechiaStudy on the Safety and Effects of Risdiplam for Adults and Children with Spinal Muscular Atrophy
BelgiumStudy of Venglustat and Imiglucerase for Adult Patients with Gaucher Disease TYPE 3
GermanyStudy on the Long-term Effects of EDG-5506 for Adults and Adolescents with Becker Muscular Dystrophy
BelgiumStudy Comparing Aficamten and Metoprolol for Adults with Symptomatic Hypertrophic Cardiomyopathy
DenmarkStudy on the Safety and Effects of BAY 2599023 (Peboctocogene Camaparvovec) for Adults with Severe Hemophilia A
BulgariaStudy of JDQ443 and Ribociclib in Combination for Adult Patients with Advanced Solid Tumors with KRAS G12C Mutation
BelgiumThe therapies tested most often in Congenital, familial and genetic disorders trials.
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