Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study on the Safety and Pharmacokinetics of Risdiplam in Infants with Spinal Muscular Atrophy
BelgiumStudy on the Effects of Pariglasgene Brecaparvovec for Adults with Glycogen Storage Disease Type Ia
The NetherlandsLong-term Study on the Safety and Effectiveness of Odevixibat for Patients with Alagille Syndrome
BelgiumStudy on How Concizumab Works for Patients with Hemophilia A or B Without Inhibitors
DenmarkStudy on Long-Term Safety of Soticlestat for Patients with Dravet or Lennox-Gastaut Syndromes
BelgiumStudy on Sotorasib for Patients with Advanced KRAS G12C-Mutated Non-Small Cell Lung Cancer and Comorbidities
DenmarkStudy on the Acceptability of Pancreas Powder for Patients with Cystic Fibrosis and Pancreatic Exocrine Insufficiency
GermanyStudy on the Effects and Safety of RO7204239 for Patients with Facioscapulohumeral Muscular Dystrophy
DenmarkStudy of Afamelanotide for Patients with Erythropoietic Protoporphyria (EPP)
BelgiumLong-term Safety Study of KVD900 for Treating Angioedema Attacks in Adolescents and Adults with Hereditary Angioedema Type I or II
AustriaStudy on the Safety and Efficacy of Viltolarsen for Boys with Duchenne Muscular Dystrophy
CzechiaStudy on the Effectiveness and Safety of Risdiplam for Children with Spinal Muscular Atrophy After Gene Therapy
GermanyThe therapies tested most often in Congenital, familial and genetic disorders trials.
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