Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study on the Safety and Effects of SQY51 for Children and Adults with Duchenne Muscular Dystrophy
FranceStudy on Marstacimab for Children with Severe Hemophilia A or B, With or Without Inhibitors
AustriaStudy on Fazirsiran for Patients with Alpha-1 Antitrypsin Deficiency-Related Liver Disease and Fibrosis
AustriaStudy on the Safety and Effects of UX701 Gene Therapy for Adults with Wilson Disease
DenmarkStudy on the Safety and Effectiveness of TransCon CNP for Children and Adolescents with Achondroplasia
AustriaStudy on Hydrochlorothiazide and Tolvaptan for Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)
AustriaStudy on the Effect of Cholestyramine on Pollutant Levels in Obese Women Eligible for Bariatric Surgery
FranceStudy on Long-Term Safety and Effectiveness of Vanzacaftor, Tezacaftor, and Deutivacaftor for Cystic Fibrosis in Patients Aged 1 Year and Older
BelgiumStudy of Microdystrophin (GNT0004) Gene Therapy for Duchenne Muscular Dystrophy in Boys Who Can Walk
BelgiumStudy on Long-Term Safety of Fazirsiran for Patients with Alpha-1 Antitrypsin Deficiency-Related Liver Disease
AustriaStudy on the Safety and Effects of Luspatercept for Children with Beta Thalassemia
GermanyStudy on the Use of Simoctocog Alfa and Emicizumab for Surgery in Patients with Severe Hemophilia A
CroatiaThe therapies tested most often in Congenital, familial and genetic disorders trials.
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