Rare diseases
Investigational molecules
Locations
374 conditions with trials, the busiest first
796 trials across Europe, recruiting first
Study on Improving Treatment for Sickle Cell Disease Patients Using Hydroxycarbamide
FranceLong-term Safety and Efficacy Study of Elivaldogene Autotemcel for Patients with Cerebral Adrenoleukodystrophy
FranceStudy on the Effects of Casimersen and Golodirsen for Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
BelgiumStudy on the Safety and Initial Effects of AAV9-CAG-coh-SGSH in Children with Sanfilippo A Syndrome
SpainStudy on Gene Therapy with Autologous Stem Cells and Drug Combination for Children with Mucopolysaccharidosis Type I Hurler Variant
ItalyStudy 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 NetherlandsThe therapies tested most often in Congenital, familial and genetic disorders trials.
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