Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study on the Effectiveness and Safety of Mitapivat for Patients with Non-Transfusion-Dependent Alpha or Beta Thalassemia
BulgariaStudy on Botulinum Toxin A and Hyoscine for Treating Drooling in Children Over 4 with Cerebral Palsy
FranceStudy on the Safety and Effects of AMT-130 for Adults with Early Huntington's Disease
PolandStudy on the Effects of ATL1102 for Nonambulatory Patients with Duchenne Muscular Dystrophy
BulgariaStudy on the Effects of Mitapivat for Patients with Transfusion-Dependent Alpha or Beta Thalassemia
BulgariaStudy on Emicizumab for Patients with Mild or Moderate Hemophilia A Without FVIII Inhibitors
BelgiumStudy of Mitapivat for Adults with Sickle Cell Disease
The NetherlandsTitle: Study of gene therapy etranacogene dezaparvovec (Hemgenix) in adult patients with moderate to severe Hemophilia B
BelgiumStudy on the Effectiveness and Safety of Fenfluramine Hydrochloride for Patients with CDKL5 Deficiency Disorder
AustriaStudy on the Effectiveness and Safety of Losmapimod for Patients with Facioscapulohumeral Muscular Dystrophy (FSHD)
DenmarkStudy on the Safety and Effects of Delandistrogene Moxeparvovec in Children Under 4 with Duchenne Muscular Dystrophy
BelgiumStudy on the Effects of Leniolisib in Children Aged 1 to 6 with Activated PI3K Delta Syndrome (APDS)
PortugalThe therapies tested most often in Congenital, familial and genetic disorders trials.
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