The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a ...

Condition: Duchenne Muscular Dystrophy

This randomized phase IIA trial studies how well antiandrogen therapy works with or without axitinib before surgery in treating patients with previously untreated prostate cancer that is known or susp ...

Condition: Metastatic Malignant Neoplasm in Lymph Node

The investigators will collect samples of sputum from healthy volunteers and patients with cystic fibrosis for the purpose of: a) purifying airway mucins for plate-based binding studies and; b) ass ...

Condition: Cystic Fibrosis

Common Variable Immunodeficiency (CVID) is a heterogeneous immune disorder characterized by a broad range of clinical manifestations. Its etiology is not yet fully understood. To gain insights into th

Childhood-onset systemic lupus erythematosus (cSLE) exhibits significant heterogeneity, leading to challenges in prognosis and treatment. This study aims to stratify cSLE patients into clinically dist

Three-dimensional (3D) human brain models have become indispensable tools to investigate neuroimmune interactions and inflammatory processes in the human central nervous system in vitro . Nevertheless