Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study of pegvaliase injections compared to diet control in adolescents aged 12-17 with phenylketonuria (PKU)
GermanyStudy on the Effectiveness and Safety of Mitapivat in Children with Pyruvate Kinase Deficiency Not Receiving Regular Blood Transfusions
FranceStudy on the Effectiveness and Safety of Avalglucosidase Alfa for Babies with Infantile-Onset Pompe Disease
BelgiumStudy of DYNE-251 safety and effectiveness in patients with Duchenne muscular dystrophy who are candidates for exon 51 skipping treatment
BelgiumStudy of Taldefgrobep Alfa for Patients with Spinal Muscular Atrophy on Stable Nusinersen and/or Risdiplam Regimen or with Onasemnogene Abeparvovec-xioi History
BelgiumStudy on Belzutifan for Treating Kidney Cancer in Patients with von Hippel-Lindau Disease
DenmarkStudy on the Effects of Fordadistrogene Movaparvovec for Patients with Duchenne Muscular Dystrophy
BelgiumStudy of G1XCGD Lentiviral Vector in Patients with X-Linked Chronic Granulomatous Disease
FranceStudy on the Safety and Effectiveness of NMD670 for Adults with Charcot-Marie-Tooth Disease Types 1 and 2
BelgiumStudy on the Safety and Effectiveness of Crinecerfont for Adults with Classic Congenital Adrenal Hyperplasia
AustriaStudy on the Effect of Human Normal Immunoglobulin on Lung Disease in Patients with Primary Antibody Deficiency
The NetherlandsStudy on Long-term Safety and Efficacy of Cipaglucosidase Alfa and Miglustat for Adults with Late-onset Pompe Disease
BelgiumThe therapies tested most often in Congenital, familial and genetic disorders trials.
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