Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study 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
FranceStudy on the Effectiveness of Atenativ for Patients with Congenital Antithrombin Deficiency Undergoing Surgery or Childbirth
AustriaStudy on the Effect of Atorvastatin in Slowing Down Arrhythmogenic Cardiomyopathy Progression in Patients with the Condition
ItalyStudy on Nepafenac and Dexamethasone for Reducing Inflammation and Swelling in Fuchs Dystrophy Patients After Corneal Transplant
NorwayStudy on the Safety and Efficacy of Avalglucosidase Alfa for Patients Aged 5 and Older with Non-Classic Pompe Disease
The NetherlandsStudy of Propranolol Treatment for Brain Hemangioblastomas in Patients with Von Hippel-Lindau Disease
FranceStudy on Estrogen Treatment (Oral vs. Transdermal) for Women with Turner Syndrome
DenmarkLong-term safety and effectiveness study of vatiquinone (oral capsules) in patients with Friedreich ataxia who have previously received this treatment
FranceStudy on Valsartan for Slowing Aortic Root Dilatation in Children and Young Adults with Marfan Syndrome and Related Conditions
PolandStudy on ABCB5+ MSCs for Recessive Dystrophic and Junctional Epidermolysis Bullosa Patients
AustriaThe therapies tested most often in Congenital, familial and genetic disorders trials.
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