Rare diseases
Investigational molecules
Locations
374 conditions with trials, the busiest first
796 trials across Europe, recruiting first
Study of anakinra safety and effectiveness given by injection under the skin in patients with cystic fibrosis
GermanyStudy on the Effects of Dimethyl Fumarate for Patients with Adrenomyeloneuropathy
SpainStudy of NIDO-361 for Patients with Spinal and Bulbar Muscular Atrophy (SBMA)
DenmarkStudy on How Concizumab Works for Patients with Hemophilia A or B with Inhibitors
CroatiaStudy on the Long-Term Safety of PTC923 (Sepiapterin) for Patients with Phenylketonuria
CzechiaStudy on the Safety and Pharmacokinetics of Risdiplam in Infants with Spinal Muscular Atrophy
BelgiumStudy on the Effects of Pariglasgene Brecaparvovec for Adults with Glycogen Storage Disease Type Ia
The NetherlandsLong-term Study on the Safety and Effectiveness of Odevixibat for Patients with Alagille Syndrome
BelgiumStudy on How Concizumab Works for Patients with Hemophilia A or B Without Inhibitors
DenmarkStudy on Long-Term Safety of Soticlestat for Patients with Dravet or Lennox-Gastaut Syndromes
BelgiumStudy on Sotorasib for Patients with Advanced KRAS G12C-Mutated Non-Small Cell Lung Cancer and Comorbidities
DenmarkStudy on the Acceptability of Pancreas Powder for Patients with Cystic Fibrosis and Pancreatic Exocrine Insufficiency
GermanyThe therapies tested most often in Congenital, familial and genetic disorders trials.
Tell us about your condition – we search every trial in Europe and connect you with the right site.
