Rare diseases
Investigational molecules
Locations
237 conditions with trials, the busiest first
741 trials across Europe, recruiting first
Study on Long-Term Safety of mRNA-3705 for Patients with Methylmalonic Acidemia (MMA)
FranceStudy on Vitamin D Deficiency Treatment in Obese Children and Adolescents Using Colecalciferol: Comparing Monthly Boluses and Daily Doses
FranceStudy on Pregnancy Rates in Women with Polycystic Ovary Syndrome Using Letrozole and Gonadotropins
SpainStudy on the Effects of Metreleptin for Patients with Partial Lipodystrophy
FranceStudy on the Safety and Effects of Cipaglucosidase Alfa and Miglustat for Children with Infantile-onset Pompe Disease
DenmarkStudy on the Effectiveness of Somatostatin Eye Drops for Patients with Moderately Severe to Severe Non-Proliferative Diabetic Retinopathy
SpainStudy on the Safety and Effects of ION356 for Patients with Pelizaeus-Merzbacher Disease
FranceStudy on Long-Term Safety and Effects of DNL310 for Patients with Mucopolysaccharidosis Type II (MPS II)
BelgiumStudy on Gene Therapy GNT0003 for Patients with Severe Crigler-Najjar Syndrome Requiring Phototherapy
FranceStudy on Semaglutide and Low Energy Diet for Obesity in Adolescents Aged 12-17
NorwayStudy on Long-Term Safety of Inclisiran for Patients with Familial Hypercholesterolemia Who Completed Previous Adolescent Studies
CzechiaStudy of ISIS 678354 for Patients with Familial Chylomicronemia Syndrome Previously Treated with Volanesorsen
SwedenThe therapies tested most often in Metabolism and nutrition disorders trials.
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