Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Study on NTLA-2002 for Adults with Hereditary Angioedema Using Ziclumeran and Lonvoguran
FranceStudy on the Safety and Effectiveness of Garetosmab for Adults with Fibrodysplasia Ossificans Progressiva
FinlandStudy on Tinlarebant for Treating Stargardt Disease in Adolescents
BelgiumStudy on the Safety and Long-Term Effects of Efavirenz for Adults and Late-Onset Juvenile Niemann-Pick Type C Disease with Cognitive Impairment
SpainStudy of Pariglasgene Brecaparvovec and Prednisolone for Adults with Glycogen Storage Disease Type Ia
DenmarkStudy of Long-term Safety and Efficacy of Fitusiran in Patients with Hemophilia A or B with or without Inhibitory Antibodies to Factor VIII or IX
DenmarkStudy of Atidarsagene Autotemcel for Treating Early Onset Metachromatic Leukodystrophy (MLD) in Patients
ItalyStudy of HORA-PDE6B's Safety in Retinitis Pigmentosa Patients with PDE6B Gene Mutations
FranceStudy on the Safety and Efficacy of Tominersen for Patients with Prodromal and Early Manifest Huntington's Disease
AustriaStudy on Selumetinib for Adults with Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas
FranceStudy of Alectinib and Platinum-Based Chemotherapy for Patients with Resected Stage Ib to IIIa ALK-Positive Non-Small Cell Lung Cancer
AustriaStudy of Long-term Safety of Tazemetostat in Patients Who Previously Participated in Tazemetostat Clinical Trials
FranceThe therapies tested most often in Congenital, familial and genetic disorders trials.
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