Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study on the Safety of VO659 for Patients with Spinocerebellar Ataxia Types 1, 3, and Huntington's Disease
DenmarkLong-term safety study of oral etavopivat tablets in children, adolescents and adults with sickle cell disease or thalassaemia who completed previous etavopivat treatment
FranceStudy on the Effectiveness of Dexamethasone in Preventing Prenatal Virilization in Female Patients with Congenital Adrenal Hyperplasia
GermanyStudy on the Effect and Safety of Autologous Mesoangioblasts for Patients with Mitochondrial Myopathy Due to m.3243A>G Mutation
The NetherlandsStudy on Reducing Treatment Frequency of Alglucosidase Alfa for Elderly Patients with Late-Onset Pompe Disease
The NetherlandsStudy on C1 Esterase Inhibitor for Treating and Preventing Hereditary Angioedema Attacks in Patients with C1-INH Deficiency
BulgariaStudy on the Effects of Injectable Gentamicin for Patients with Moderate to Severe Congenital Ichthyosis
FranceStudy on Bevacizumab for Patients with Symptomatic Cerebral Arteriovenous Malformations
FranceStudy on Dapagliflozin for Slowing Kidney Disease in Adolescents and Young Adults with Alport Syndrome
GermanyLong-term safety study of rebisufligene etisparvovec gene therapy in patients previously treated for Mucopolysaccharidosis type IIIA
SpainStudy on the Safety and Effectiveness of Rebisufligene Etisparvovec for Patients with Mucopolysaccharidosis Type IIIA
SpainStudy on the Early Use of Risdiplam for Children with Spinal Muscular Atrophy After Gene Therapy
GermanyThe therapies tested most often in Congenital, familial and genetic disorders trials.
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