Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study on Tildacerfont to Reduce Glucocorticoid Use in Adults with Classic Congenital Adrenal Hyperplasia
DenmarkStudy to Evaluate the Effectiveness of Spesolimab in Treating Patients with Netherton Syndrome
AustriaStudy on the Effects of Dimethyl Fumarate for Patients with Friedreich's Ataxia
ItalyStudy on Metreleptin for Children Under 6 with Generalized Lipodystrophy and Related Diabetes or High Triglycerides
BelgiumStudy on the Effects of Ecopipam Hydrochloride for Children and Adolescents with Tourette's Disorder
BulgariaStudy on the Efficacy and Safety of Fidanacogene Elaparvovec Gene Therapy in Adult Males with Moderate to Severe Hemophilia B
BelgiumStudy on Fitusiran for Preventing Bleeding in Males Aged 12 and Older with Severe Hemophilia A or B, with or without Inhibitors
FranceStudy on Luspatercept for Treating Anemia in Adults and Adolescents with Alpha-Thalassemia
GreeceStudy on Tadekinig Alfa for Patients with NLRC4 Mutation and XIAP Deficiency
GermanyStudy on Long-Term Safety and Efficacy of Gene Therapy for Leukocyte Adhesion Deficiency-I Using RP-L201 in Patients with LAD-I
SpainStudy on the Safety of DS-2325a for Patients with Netherton Syndrome
FranceStudy on Crovalimab for Preventing Painful Episodes in Sickle Cell Disease Patients
FranceThe therapies tested most often in Congenital, familial and genetic disorders trials.
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