Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Safety and Efficacy of TSRA-196 (rnaivt9315 and rnacs24757) in Adults with Lung or Liver Disease due to Alpha‑1 Antitrypsin Deficiency
IrelandSafety, Tolerability, Pharmacokinetics and Pharmacodynamics of DNL952 in Adults with Late‑Onset Pompe Disease
GermanyEvaluation of NXT007 versus Emicizumab Prophylaxis in Patients with Hemophilia A
AustriaA Phase III study of NXT007 compared with simoctocog alfa prophylaxis in patients with Hemophilia A without inhibitors
AustriaEfficacy and safety of human alpha1‑proteinase inhibitor weekly infusions in adults with alpha1 antitrypsin deficiency–related emphysema
Denmark
BelgiumDuchenne Muscular Dystrophy in Patients with Gene Mutations Amenable to Exon 44 Skipping Treated with Delpacibart Zotadirsen
BelgiumLerodalcibep for Heterozygous Familial Hypercholesterolemia in Children and Adolescents 6 to 17 Years Old
NorwayBMN 333 Versus Vosoritide in Children With Achondroplasia
FranceA study to evaluate the efficacy and safety of DYNE-101 in patients with Myotonic Dystrophy Type 1
BelgiumBelgiumA study to evaluate the safety of MRM-3379 in men with Fragile X Syndrome
SpainThe 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.
