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This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children < 2 years of age genetically diagnosed with …
Using a focused implementation research framework, the EPIS (Exploration, Preparation, Implementation and Sustainment) model in a type-2 hybrid design, the study will be conducted in 3 phases: 1) A pre-implementation phase that will use the Exploration and Preparation domains of EPIS to: a) explore barriers and facilitators of Clean Fuel- …
This is an observational clinical trial, aiming to investigate whether the ctDNA dynamics could predict early response to ICIs in patients with advanced-stage cancer. Moreover, conventional tumor markers PD-L1, TMB and MSI are to be investigated for their combined prognostic values in ICI treatment.
The purpose of this study is to evaluate the clinical utility of neurophysiological measurements of ECAP-controlled closed-loop SCS (i.e., neural panel metrics) to guide treatment of chronic pain of the trunk and/or limbs.
The study's purpose is to see if the drug, abemaciclib, is safe and effective when given with other drugs to kill cancer cells. The study is open to children and young adults with solid tumors, including neuroblastoma, that did not respond or grew during other anti-cancer treatment. For each participant, …
The goal of this study is to use a clinical micro-ultrasound to systematically image the prostate before biopsy or surgery. The images from the ultrasound system will be saved and compared to other imaging modalities and pathology in order to develop better tools.
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