Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study on ATX-01 for Adults Aged 18-64 with Myotonic Dystrophy Type 1 (DM1)
FranceStudy on the Effects of Bisoprolol and Verapamil in Patients with Non-Obstructive Hypertrophic Cardiomyopathy
DenmarkStudy on the Effects of Dronabinol and Palmidrol for Adults with Tourette Syndrome
GermanyStudy on the Safety and Effects of Efpegerglucagon in Children Aged 2 and Older with Congenital Hyperinsulinism
GermanyStudy of doxecitine and doxribtimine oral solution for adult patients with Thymidine Kinase 2 (TK2) Deficiency
SpainStudy on the Effects of Exagamglogene Autotemcel for Patients with Transfusion-Dependent Beta-Thalassemia or Severe Sickle Cell Disease
GermanyStudy on Patritumab Deruxtecan and Zirconium (89Zr) Patritumab Deruxtecan for Patients with Advanced EGFR Mutation Positive Lung Cancer
The NetherlandsStudy on the Safety and Effects of NXT007 for Patients with Severe or Moderate Hemophilia A
ItalyStudy on Long-Term Safety of mRNA-3705 for Patients with Methylmalonic Acidemia (MMA)
FranceStudy on the Effects of Sirolimus and Drug Combination for Children Under 4 Months with Tuberous Sclerosis Complex
AustriaTestosterone Treatment for Boys with Klinefelter Syndrome: A Study on Body Composition Changes During Puberty
DenmarkStudy on the Safety and Effects of Cipaglucosidase Alfa and Miglustat for Children with Infantile-onset Pompe Disease
DenmarkThe therapies tested most often in Congenital, familial and genetic disorders trials.
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