Rare diseases
Investigational molecules
Locations
453 conditions with trials, the busiest first
1 389 trials across Europe, recruiting first
Study of pegvaliase injections compared to diet control in adolescents aged 12-17 with phenylketonuria (PKU)
GermanyStudy on the Use of PD L 506 and Aminolevulinic Acid Hydrochloride for New Glioblastoma in Adults
GermanyStudy on Long-Term Safety and Efficacy of Ocrelizumab for Patients with Multiple Sclerosis
AustriaStudy on the Effectiveness and Safety of Batoclimab for Adults with Generalized Myasthenia Gravis
GermanyStudy on Ofatumumab for Patients with Relapsing Multiple Sclerosis Transitioning from Fumarate-Based Therapies or Fingolimod
BulgariaStudy on the Effectiveness and Safety of Ofatumumab and Siponimod Compared to Fingolimod in Children with Multiple Sclerosis
AustriaStudy 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 Effectiveness and Safety of AC102 Injection vs. Oral Prednisolone for Patients with Sudden Hearing Loss
AustriaStudy on the Effects of Fordadistrogene Movaparvovec for Patients with Duchenne Muscular Dystrophy
BelgiumThe therapies tested most often in Nervous system disorders trials.
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