Rare diseases
Investigational molecules
Locations
374 conditions with trials, the busiest first
796 trials across Europe, recruiting first
Study on Metoprolol for Preventing Heart Disease in Boys with Duchenne Muscular Dystrophy Aged 8-17
PolandStudy on the Effects of Metformin on Cognitive Function in Patients with Huntington's Disease
SpainStudy on Rigosertib Sodium for Patients with Recessive Dystrophic Epidermolysis Bullosa and Advanced Squamous Cell Carcinoma
AustriaStudy on the Effects of BT200 (Rondaptivon Pegol) for Patients with Type 2B von Willebrand Disease
AustriaStudy on Mesalamine to Prevent Colorectal Cancer in Patients with Lynch Syndrome
DenmarkStudy on the Safety and Tolerability of WVE-006 for Patients with Alpha-1 Antitrypsin Deficiency (AATD) Pi*ZZ
FinlandStudy on the Effects of TTI-0102 for Patients with MELAS Syndrome
FranceStudy on Leuprorelin Acetate for Slowing Disease Progression in Premenopausal Women with Severe Polycystic Liver Disease
The NetherlandsLong-term Safety Study of Cholic Acid for Patients with Bile Acid Synthesis Defects
The NetherlandsStudy Comparing Alectinib and Crizotinib for Patients with ALK-Positive Advanced Non-Small Cell Lung Cancer
ItalyStudy on the Long-Term Safety of GTX-102 for Patients with Angelman Syndrome
FranceStudy on the Safety and Tolerability of Oral Dersimelagon for Patients with Erythropoietic Protoporphyria or X-Linked Protoporphyria
BulgariaThe therapies tested most often in Congenital, familial and genetic disorders trials.
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