Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study of MK-0616 for Adults with Familial Hypercholesterolemia
CzechiaStudy on the Effects of Deucravacitinib for Adults with Inflammatory Skin Conditions (Epidermolysis Bullosa Simplex and Congenital Ichthyoses)
FranceStudy on the Safety and Effectiveness of CRN04894 for Patients with Congenital Adrenal Hyperplasia
GermanyStudy on VX-522 and Ivacaftor for Adults with Cystic Fibrosis Unresponsive to CFTR Modulator Therapy
BelgiumStudy on the Effects of Romosozumab and Bisphosphonates in Children and Adolescents with Osteogenesis Imperfecta
AustriaLong-term Safety Study of Mavacamten for Adults with Hypertrophic Cardiomyopathy from Previous Trials
BelgiumStudy on the Safety and Effects of STAR-0215 for Adults with Hereditary Angioedema
BulgariaStudy on the Safety and Effects of Garadacimab for Preventing Hereditary Angioedema in Children Aged 2 to 11
GermanyStudy on the Safety and Effectiveness of SXR1096 Cream for Patients with Netherton Syndrome
AustriaStudy on Valbenazine for Treating Dyskinesia in Children and Adults with Cerebral Palsy
BelgiumStudy on the Safety and Efficacy of Cipaglucosidase Alfa and Miglustat for Children with Late-onset Pompe Disease
GermanyGene Therapy Study for Patients with Limb-Girdle Muscular Dystrophy Using GNT0006
DenmarkThe therapies tested most often in Congenital, familial and genetic disorders trials.
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