Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study on Emicizumab and Drug Combination for Patients with Haemophilia A and FVIII Inhibitors
BulgariaStudy on the Safety and Effects of ION582 for Patients with Angelman Syndrome
FranceStudy on the Safety and Effects of Infusing Modified CD34+ Cells for Patients with Fanconi Anemia Subtype A
SpainStudy on Early Treatment of Patent Ductus Arteriosus with Paracetamol in Extremely Low Birth Weight Infants
CzechiaStudy on Rocuronium Bromide for Neuromuscular Block in Infants with Craniosynostosis During Surgery
HungaryStudy on the Effects of Mexiletine for Treating Myotonic Dystrophy Types 1 and 2 in Patients
BelgiumLong-Term Safety and Efficacy Study of Givinostat for Patients with Duchenne Muscular Dystrophy Previously Treated with Givinostat
BelgiumStudy of zongertinib tablets in patients with advanced solid tumors that have HER2 gene changes
BelgiumStudy on Long-Term Use of Infigratinib for Children with Achondroplasia
FranceStudy on Long-Term Safety and Pregnancy Outcomes of Pariglasgene Brecaparvovec in Patients with Glycogen Storage Disease Type Ia (GSDIa)
DenmarkStudy on the Safety and Effects of Pegunigalsidase Alfa for Children and Adolescents with Fabry Disease
AustriaStudy of VX-121, Tezacaftor, and Deutivacaftor for Children Aged 1-11 with Cystic Fibrosis
BelgiumThe therapies tested most often in Congenital, familial and genetic disorders trials.
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