Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
A study to evaluate the safety and effectiveness of pegtibatinase in patients aged 12 to 65 years with classical homocystinuria.
BelgiumA study on the long-term safety and effectiveness of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B
FranceLong‑Term Safety and Efficacy of Infigratinib in Children with Hypochondroplasia
NorwayA Phase 3 Study of Fenfluramine Hydrochloride for Safety and Efficacy in Patients with Rett Syndrome
BelgiumA study to evaluate the safety and effectiveness of AAVB-039 in patients with Stargardt disease
BelgiumA study to evaluate the effectiveness of bleomycin sulfate for treating patients with low-flow vascular malformations using electrosclerotherapy.
SloveniaA study to evaluate the safety and tolerability of tinlarebant in patients with Stargardt disease
BelgiumA study to evaluate the safety and effectiveness of surlorian in adults with RYR1-related myopathy
FranceStudy to evaluate the effect of omaveloxolone on mitochondrial function in patients with Friedreich's ataxia
CzechiaUsing [18F]DED PET imaging to study brain changes in patients with Alzheimer's disease, CADASIL, and other forms of dementia.
SpainA study to evaluate the long-term safety and effectiveness of BMN 351 in patients with Duchenne muscular dystrophy amenable to exon 51 skipping.
ItalyA Phase 3 study to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome
GermanyThe therapies tested most often in Congenital, familial and genetic disorders trials.
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