Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study on Dantrolene Sodium for Treating Darier Disease in Affected Patients
SwedenStudy on Mavorixafor for Treating WHIM Syndrome in Patients
DenmarkStudy on the Safety of Allogeneic Adipose Tissue-Derived Mesenchymal Stromal Cells for Patients with Epidermolysis Bullosa
CzechiaStudy on the Effects and Safety of ZYN002 Gel for Children, Adolescents, and Young Adults with Fragile X Syndrome
IrelandStudy of Trastuzumab, Pertuzumab, and Chemotherapy Combination with Response-Guided Treatment for Patients with HER2-Positive Breast Cancer
The NetherlandsStudy of Osimertinib for Patients with EGFR-Mutated Non-Small Cell Lung Cancer
LithuaniaStudy of BDC-1001 and Nivolumab for Patients with Advanced HER2-Positive Solid Tumors
FranceStudy on the Effectiveness and Safety of DNL310 vs Idursulfase for Children and Young Adults with Mucopolysaccharidosis Type II
BelgiumA Phase 2 Study to Evaluate Safety and Effectiveness of Omaveloxolone (RTA 408) in Patients with Friedreich's Ataxia
AustriaStudy on the Long-Term Safety of STAR-0215 for Adults with Hereditary Angioedema
BulgariaStudy on the Effect of Food on Selumetinib in Adolescents with Neurofibromatosis Type 1 Plexiform Neurofibromas
PolandLong-Term Safety Study of Everolimus for Patients with Tuberous Sclerosis Complex and Refractory Seizures
PolandThe therapies tested most often in Congenital, familial and genetic disorders trials.
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