Rare diseases
Investigational molecules
Locations
Study on the Effectiveness and Safety of Vosoritide for Children with Hypochondroplasia
FranceStudy on BI 1015550 for Improving Lung Function in Patients with Progressive Fibrosing Interstitial Lung Diseases
FranceStudy on Anifrolumab and Phototherapy for Adults with Progressive Vitiligo
FranceStudy of Efgartigimod PH20 SC for Adults with Thyroid Eye Disease
FranceStudy of Sotatercept for Newly Diagnosed Intermediate- and High-risk Pulmonary Arterial Hypertension Patients
FranceLong-term Study of Sotatercept for Adults with Pulmonary Arterial Hypertension (PAH)
FranceStudy of Berubicin versus Lomustine in adult patients with recurrent Glioblastoma Multiforme after first-line treatment failure
FranceStudy of Avalotcagene Ontaparvovec for Patients Aged 12 and Older with Late-onset Ornithine Transcarbamylase Deficiency
FranceStudy on Long-Term Safety and Efficacy of Cebsulfase Alfa for Children with Late Metachromatic Leukodystrophy Receiving Intrathecal Enzyme Replacement
FranceStudy on Mepolizumab and Drug Combination for Patients with Eosinophilic Granulomatosis with Polyangiitis
FranceStudy on Glenzocimab for Patients with Acute Ischemic Stroke Undergoing Mechanical Thrombectomy
FranceStudy of Setmelanotide for Patients with Obesity Due to Specific Genetic Variants
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