Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study on the Safety and Effects of SPK-3006 for Adults with Late-Onset Pompe Disease
GermanyStudy on Sildenafil for Adults with Single Ventricle Heart Condition Post-Fontan Surgery
PolandStudy on the Effectiveness of INCB000928 for Patients with Fibrodysplasia Ossificans Progressiva
FranceStudy on Gene Therapy for Severe Combined Immunodeficiency (SCID) Using ARTEGENE in Patients with Artemis Gene Mutations
FranceImaging Study with 89Zr-Bevacizumab for Predicting Treatment Effects in Patients with NF2-Related Schwannomatosis
The NetherlandsStudy on Early Oxybutynin Treatment for Boys with Posterior Urethral Valves
FranceStudy on Improving Bone Health in Children with Cystic Fibrosis Using Cholecalciferol
CzechiaStudy on the Safety of Inhaled Teicoplanin for Treating Staphylococcus aureus Infections in Cystic Fibrosis Patients
ItalyStudy on the Safety and Effects of Givinostat in Young Children with Duchenne Muscular Dystrophy (Ages 2 to Less Than 6 Years)
BelgiumLong-Term Safety and Efficacy Study of Mexiletine Hydrochloride for Patients with Myotonic Dystrophy Type 1 and Type 2
BelgiumStudy on Sclerotherapy for Hydrocele and Spermatocele Using Lauromacrogol 400 and Ethanol in Adult Patients
SwedenStudy of KK8123 for Adults with X-linked Hypophosphatemia
FranceThe therapies tested most often in Congenital, familial and genetic disorders trials.
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