Rare diseases
Investigational molecules
Locations
374 conditions with trials, the busiest first
796 trials across Europe, recruiting first
Study on Sirolimus and Prednisolone for Treating Superficial Arteriovenous Malformations in Patients
FranceStudy on the Effectiveness of Deflazacort in Treating LMNA-related Congenital Muscular Dystrophy in Adults and Children
ItalyStudy of GBT021601 for Adults and Children with Sickle Cell Disease
BelgiumStudy Comparing Custodiol-N and Custodiol for Heart Surgery in Children with Congenital Heart Defects
GermanyStudy on Trametinib for Patients with Neurofibromatosis Type 1 and Symptomatic Plexiform Neurofibromas
The NetherlandsStudy on the Safety of Sirolimus for Children with Familial Adenomatous Polyposis
FranceStudy on the Safety and Effectiveness of Delandistrogene Moxeparvovec for Patients with Duchenne Muscular Dystrophy
BelgiumStudy 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
BelgiumThe therapies tested most often in Congenital, familial and genetic disorders trials.
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