Sanfilippo syndrome Type A or mucopolysaccharidosis type IIIA (MPS IIIA) is a rare genetic disorder caused by the deficiency of the lysosomal enzyme sulfamidase. Lack of this enzyme causes buildup of large sugar molecules called glycosaminoglycans (GAGs) in the lysosomes which leads to cellular dysfunction. The buildup of GAGs causes significant damage to the body and brain. This study is a multicenter, Phase 1/2 study to assess the safety, tolerability, and efficacy of they study drug DNL126, an enzyme replacement therapy, to provide the missing enzyme to the body. Up to 28 participants with MPS IIIA will participate in the study across multiple study sites .

To provide a teaching tool to dermatology residents and assess its efficacy

To determine the helpfulness of using images of brain anatomy to educate patients about their cognitive disorders.

The purpose of this study is to figure out the best way to work with providers and staff who treat pregnant people with very high blood pressure. We will send a nurse to 20 clinics that provide prenatal care in the community. For 12 months, the nurse will train and coach staff on implementing guidelines for addressing severe blood pressure. Some of the training will use patient actors who need their blood pressure measured, or who come to the clinic with very high blood pressure. The nurse will observe the clinic team as they interact with the "pretend" patient, and talk to them afterwards about ways to improve care. We will collect data before, during, and after the nurse has worked in the clinic. Information from 1) observations, and 2) patient charts of people who have already delivered, will tell us if our efforts in the clinics made a difference.

Compare the ability of crowd-sourced amateurs in identifying and labeling complex constructs (here, the 4 Ps of Marketing) in user-generated content (here, Tweets) with that of generative AI (here, ChatGPT4) . Can generative AI match or even exceed crowd-sourced labels?

The purpose of the study is to understand why it is the case that authors do not submit reproducible research compendia even when a policy requires it (and policy guidance explains how). To do so, the study looks toward the specific mechanisms that inhibit and/or facilitate execution of data management tasks that support reproducibility. Identifying these mechanisms can inform the design of interventions that better equip researchers to produce and share high-quality research compendia. Such interventions may reduce the amount of time and labor currently required for current data verification workflows, which in turn may make journal-based data policies more feasible for a greater number of journals.

To understand barriers to access treatment and knowledge of oral health among foster families in North Carolina

To demonstrate efficacy of Bexsero in prevention of urogenital and/or anorectal gonococcal infection. To estimate efficacy of Bexsero in prevention of overall gonococcal infection and by anatomical site (urogenital, anorectal or pharyngeal). To assess safety of Bexsero.

The purpose of this research study is to measure whether people are biased in terms of how quickly or slowly they respond to members of different social groups.

The purpose of this study is to understand the perspectives and experiences of teachers working in U.S. schools in Latin America. We hope to better understand how teachers came to their positions, explore the impact of lived experiences in teaching, and understand how biliteracy, bilingualism, cross-cultural appreciation, and critical consciousness is operationalized in schools.