Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study on Aficamten for Adults with Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy
DenmarkLong-term Safety Study of Etranacogene Dezaparvovec in Adult Men with Hemophilia B
BelgiumStudy on the Effects of Gemlapodect for Adults and Adolescents with Tourette Syndrome
BelgiumStudy of Empagliflozin for Patients with Autosomal Dominant Polycystic Kidney Disease
GermanyStudy on the Effects and Safety of INZ-701 for Children with ENPP1 Deficiency
FranceStudy on the Effects and Safety of Emicizumab for Infants with Hemophilia A (Ages Birth to 12 Months)
AustriaStudy on the Safety and Effects of INZ-701 for Infants with ENPP1 or ABCC6 Deficiency
SpainStudy on the Effects of Dersimelagon Phosphate for Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria
BulgariaSafety Study of Damoctocog Alfa Pegol for Children Aged 7 to 11 with Severe Hemophilia A
ItalyStudy of adagrasib in elderly patients (70 years or older) or patients with poor performance status who have KRAS G12C mutation-positive non-small cell lung cancer
BelgiumStudy on the Effectiveness and Safety of Infigratinib for Children Aged 3 to 17 with Achondroplasia
FranceStudy on Oral PHA-022121 for Preventing Angioedema Attacks in Patients with Hereditary Angioedema
AustriaThe therapies tested most often in Congenital, familial and genetic disorders trials.
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