Rare diseases
Investigational molecules
Locations
374 conditions with trials, the busiest first
796 trials across Europe, recruiting first
Study on Obeticholic Acid for Children with Biliary Atresia After Kasai Procedure
BelgiumStudy on ALXN1850 and Asfotase Alfa for Children Aged 2-12 with Hypophosphatasia Previously Treated with Asfotase Alfa
FranceStudy on the Safety of LY3884961 for Patients with Type 1 Gaucher Disease
GermanyStudy on the Effectiveness and Safety of Exa-cel for Adolescents and Adults with Severe Sickle Cell Disease
FranceStudy on MK-0616 for Reducing Heart Disease Events in Patients with Atherosclerotic Cardiovascular Disease
DenmarkStudy on the Safety and Feasibility of Using Autologous Oral Mucosa-Derived Keratinocytes and Fibroblasts for Treating Cleft Palate Patients
SpainStudy on the Effects of Dirocaftor, Posenacaftor, and Nesolicaftor for Adults with Cystic Fibrosis
BelgiumStudy on Joint Health in Hemophilia A and B Patients Using Efmoroctocog Alfa or Eftrenonacog Alfa
BulgariaStudy on Gene Therapy with AAVrh10 for Infants with Krabbe Disease Undergoing Stem Cell Transplantation
SpainStudy of Cytarabine and Daunorubicin for Treating Myeloid Leukemia in Children with Down Syndrome
AustriaStudy on Spironolactone for Patients with Arrhythmogenic Right Ventricular Dysplasia (ARVD)
FranceA Study Testing How Well BAY 3401016 Works and How Safe It Is in Adults Aged 18 to 45 Years with Alport Syndrome
CzechiaThe therapies tested most often in Congenital, familial and genetic disorders trials.
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