Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study on Testosterone and Isopropyl Myristate for Women with Turner Syndrome
DenmarkStudy of glycerol phenylbutyrate (oral liquid) for treatment of fatigue in patients with pyruvate dehydrogenase deficiency
FranceLong-term Safety and Efficacy Study of Etuvetidigene Autotemcel for Patients with Wiskott-Aldrich Syndrome
ItalyStudy on the Effectiveness and Safety of Sepofarsen for Patients with Leber Congenital Amaurosis Due to a Specific Genetic Mutation
BelgiumStudy on Inclisiran, Alirocumab, and Rosuvastatin for Children with Familial Hypercholesterolemia
PolandStudy on the Safety and Effectiveness of CRN04894 for Patients with Congenital Adrenal Hyperplasia
AustriaStudy of N-Acetyl-L-Leucine in Patients with Ataxia-Telangiectasia: A Phase III Trial
GermanyStudy on the Effects of TCP-25 Gel for Wound Healing in Patients with Epidermolysis Bullosa
FranceStudy on Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa Using Skin Grafts with Genetically Corrected Autologous Keratinocytes and Fibroblasts
FranceStudy of SGT-003 Gene Therapy for Safety and Tolerability in Walking Males with Duchenne Muscular Dystrophy
ItalyStudy on the Effects of Pitolisant Hydrochloride for Patients with Prader-Willi Syndrome
BelgiumStudy on the Safety and Effectiveness of Etranacogene Dezaparvovec Gene Therapy for Adults with Severe or Moderately Severe Hemophilia B and AAV5 Antibodies
BulgariaThe therapies tested most often in Congenital, familial and genetic disorders trials.
Tell us about your condition – we search every trial in Europe and connect you with the right site.
