Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study on the Effects of EDG-5506 for Adults and Adolescents with Becker Muscular Dystrophy
BelgiumLong-Term Study on the Safety of Avalotcagene Ontaparvovec for Adults with Late-Onset Ornithine Transcarbamylase Deficiency
FranceStudy on the Effects of Risdiplam in Adults with Spinal Muscular Atrophy Types 2 or 3 or with Up to 4 SMN2 Gene Copies
BelgiumStudy on the Safety and Effects of PBF-999 for Patients with Prader-Willi Syndrome
SpainStudy on Trehalose Dihydrate for Treating Spinocerebellar Ataxia in Adults
GermanyStudy on the Effect of Famotidine in Reducing P-selectin Levels in Children with Sickle Cell Disease
FranceStudy on Long-Term Safety of Gene Therapy for Pyruvate Kinase Deficiency Using Autologous CD34+ Cells in Adults and Children
SpainLong-Term Safety Study of HepaStem for Patients with Urea Cycle Disorders, Crigler-Najjar Syndrome, and Fibroinflammatory Liver Diseases
BelgiumStudy on Physical Activity and Joint Health in Patients Aged 12 and Older with Severe Hemophilia A Treated with Efanesoctocog Alfa for 24 Months
AustriaStudy on the Safety and Effectiveness of Somapacitan for Children with Short Stature Due to Noonan Syndrome, Turner Syndrome, Small for Gestational Age, or Idiopathic Short Stature
The NetherlandsStudy on the Effects of Oral N-Acetylcysteine for Patients with Retinitis Pigmentosa
AustriaStudy on the Effects of Tildacerfont and Hydrocortisone in Adults with Classic Congenital Adrenal Hyperplasia
DenmarkThe therapies tested most often in Congenital, familial and genetic disorders trials.
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