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Autologous hematopoietic stem cell gene therapy in RAG-deficient severe combined immunodeficiency: pre-phase study

The purpose of this study is to evaluate the feasibility and efficiency of transducing freshly obtained CD34+ HSC from either mobilized peripheral blood or bone marrow of healthy donors using lentiviral SIN vectors encoding codon-optimized human…

PsA digital phenotyping and inflammation drivers study

Primary objectives:  To develop and internally validate a novel and interpretable machine learning model for detecting flare in PsA patients using integrated accelerometer data, keystroke dynamics and screen time metrics (i.e., digital…

A European multicenter, observational study to understand how the use of lebrikizumab affects health-related well-being and disease symptoms in patients with moderate-to-severe atopic dermatitis (ADTrust).

Primary objective: To assess the long-term effect of treatment with lebrikizumab on the overall well-being in patients with moderate-to-severe AD using the WHO-5 questionnaire. Secondary objectives: To assess the clinical effectiveness of…