Rare diseases
Investigational molecules
Locations
374 conditions with trials, the busiest first
796 trials across Europe, recruiting first
Study 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
CroatiaStudy on the Use of Simoctocog Alfa for Women and Girls with Hemophilia A Undergoing Surgery
FinlandStudy on Early Use of Candesartan vs Placebo for People with Genetic Risk of Dilated Cardiomyopathy
The NetherlandsStudy on Tocilizumab for Treating Acute Chest Syndrome in Children and Adults with Sickle Cell Disease
FranceThe therapies tested most often in Congenital, familial and genetic disorders trials.
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