Rare diseases
Investigational molecules
Locations
374 conditions with trials, the busiest first
796 trials across Europe, recruiting first
Study on Enalapril for Adults with Painful Venous Malformations
NorwayStudy on the Effectiveness and Safety of Apremilast for Patients Aged 6 and Older with Generalized Epidermolysis Bullosa Simplex
FranceStudy on the Effectiveness of Concizumab for Children Under 12 with Hemophilia A or B, With or Without Inhibitors
BulgariaStudy on the Effects of Metformin for Improving Muscle Function in Patients with Myotonic Dystrophy Type 1
FranceStudy on the Safety and Effects of Sparsentan for Children with Proteinuric Kidney Diseases
GermanyLong-term Safety and Efficacy Study of Lovotibeglogene Autotemcel Gene Therapy for Patients with Sickle Cell Disease
FranceStudy on the Effectiveness of Methylphenidate for Children and Adolescents with KBG Syndrome
The NetherlandsStudy on the Long-Term Safety of Aficamten (CK-3773274) for Patients with Symptomatic Hypertrophic Cardiomyopathy
CzechiaStudy on Personalized Dosing of Emicizumab for Patients with Congenital Hemophilia A
The NetherlandsStudy on ATX-01 for Adults Aged 18-64 with Myotonic Dystrophy Type 1 (DM1)
FranceStudy on the Effects of Bisoprolol and Verapamil in Patients with Non-Obstructive Hypertrophic Cardiomyopathy
DenmarkStudy on the Effects of Dronabinol and Palmidrol for Adults with Tourette Syndrome
GermanyThe therapies tested most often in Congenital, familial and genetic disorders trials.
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