Rare diseases
Investigational molecules
Locations
237 conditions with trials, the busiest first
741 trials across Europe, recruiting first
Study of Efgartigimod for Adults with Thyroid Eye Disease
BelgiumStudy on INZ-701 for Improving Survival in Infants with ENPP1 Deficiency
FranceStudy on the Effects of ARO-APOC3 for Adults with Familial Chylomicronemia Syndrome
AustriaStudy of Avalotcagene Ontaparvovec for Patients Aged 12 and Older with Late-onset Ornithine Transcarbamylase Deficiency
FranceLong-term Safety Study of INZ-701 for Patients with ENPP1 and ABCC6 Deficiencies, Including Pseudoxanthoma Elasticum and Generalized Arterial Calcification of Infancy
FranceStudy on Levothyroxine and Sildenafil for Improving Erectile Function in Patients with Subclinical Hypothyroidism and Erectile Dysfunction
SpainStudy on Gene Therapy with Atidarsagene Autotemcel for Patients with Metachromatic Leukodystrophy
ItalyStudy of SPI-62 for Treating ACTH-Dependent Cushing's Syndrome in Patients with Cushing's Disease or Ectopic ACTH/CRH Secretion
BulgariaStudy on Long-Term Safety and Efficacy of Cebsulfase Alfa for Children with Late Metachromatic Leukodystrophy Receiving Intrathecal Enzyme Replacement
CzechiaStudy of Insulin Treatment for Blood Glucose Control in Adult Critically Ill Patients with Stress Hyperglycemia
BelgiumStudy on the Effects of Ertugliflozin and Sodium Intake on Blood Pressure in Overweight Adults with Type 2 Diabetes
The NetherlandsStudy of SPI-62 for Treating Cushing's Syndrome in Patients with Benign Adrenal Tumors
RomaniaThe therapies tested most often in Metabolism and nutrition disorders trials.
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