Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study on the Safety and Effectiveness of AGTC-501 for Men with X-linked Retinitis Pigmentosa
SpainStudy on Sirolimus for Treating Cervico-facial Lymphatic Malformations in Patients with Poor Prognosis
FranceStudy on Mavacamten for Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy
AustriaStudy on the Safety of ATA-200 Gene Therapy for Patients with Limb-Girdle Muscular Dystrophy Type R5
FranceStudy of ALXN1850 for Children Aged 2 to 12 with Hypophosphatasia Who Have Not Previously Received Asfotase Alfa Treatment
BelgiumStudy 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
BelgiumThe therapies tested most often in Congenital, familial and genetic disorders trials.
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