Rare diseases
Investigational molecules
Locations
374 conditions with trials, the busiest first
796 trials across Europe, recruiting first
Study on the Safety and Efficacy of RO7204239 and Risdiplam for Patients with Spinal Muscular Atrophy
BelgiumStudy of Exagamglogene Autotemcel (CTX001) for Treatment of Severe Sickle Cell Disease Using Modified Stem Cells
BelgiumStudy on the Safety and Efficacy of Delandistrogene Moxeparvovec and Imlifidase for Patients with Duchenne Muscular Dystrophy with Pre-existing Antibodies
SpainStudy on Long-term Safety and Efficacy of Elexacaftor, Tezacaftor, and Ivacaftor in Children and Adults with Cystic Fibrosis Aged 2 Years and Older
GermanyStudy on the Safety and Effectiveness of BOOST Cells for Treating Severe Osteogenesis Imperfecta in Children Before and After Birth
The NetherlandsStudy of JDQ443, TNO155, and Tislelizumab for Patients with Advanced Solid Tumors with KRAS G12C Mutation
BelgiumStudy on ECUR-506A and ECUR-506D for Male Infants Under 9 Months with Neonatal Onset Ornithine Transcarbamylase Deficiency
SpainStudy on Basimglurant for Children, Adolescents, and Young Adults with Tuberous Sclerosis Complex-Related Seizures
ItalyStudy on the Effects of Satralizumab in Children and Adolescents with Duchenne Muscular Dystrophy
DenmarkStudy of Trientine Dihydrochloride (Cufence) to Evaluate Its Effects and Safety in Patients with Wilson's Disease
DenmarkStudy on Alpelisib and Fulvestrant for Advanced Breast Cancer in Patients with PIK3CA Mutation and Hormone-Receptor Positive, HER2 Negative Tumors
The NetherlandsStudy on Long-Term Safety of Ruxolitinib, Panobinostat, and Siremadlin for Patients Continuing Treatment from Previous Studies
GermanyThe therapies tested most often in Congenital, familial and genetic disorders trials.
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