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Medical center supports patient advocacy.

At Hurley Medical Center, patient advocates enjoy an amiable relationship with administrators and are considered a valuable management information resource.

Consumer Behavior

The 9th International RASopathies Symposium.

The RASopathies are a group of congenital disorders with overlapping clinical manifestations that are caused by pathogenic germline or early somatic variants that result in the hyperactivation of the RAS/mitogen-activated protein kinase (MAPK) signaling pathway. Given the heterogeneous clinical presentations of these disorders that involve abnormalities across multiple organ systems, multidisciplinary clinical management and progress in scientific research are essential for optimal patient diagnosis and care. The 9th International RASopathies Symposium, a biennial meeting, was organized by the patient advocacy group RASopathies Network and showcased recent discoveries, case studies, and advances in preclinical research. Participants, who included scientists, clinicians, industry representatives, patients, and family advocates, explored knowledge gaps, innovative clinical approaches, and lived experiences of individuals with a RASopathy. Sessions centered around organ systems were introduced with a patient perspective to highlight the burden of disease, continued with presentations from established and early-career investigators. Overall, the RASopathies Symposia serve as a catalyst for sustained community collaboration focused on enhancing patient health and accelerating the translation of discoveries into effective treatments.

Humans

289th ENMC international workshop: assessing and managing emerging AAV related toxicities after gene therapy for neuromuscular disorders, 26 - 28 September 2025, Hoofddorp, The Netherlands.

Adeno-associated virus (AAV) mediated gene therapies has emerged as a potentially transformative treatment approaches for neuromuscular disorders, with two FDA-approved products now in widespread clinical use: onasemnogene abeparvovec (Zolgensma) for spinal muscular atrophy and delandistrogene moxeparvovec-rokl (Elevidys) for Duchenne Muscular Dystrophy. However, severe and occasionally fatal adverse events affecting vital organs, including the blood, liver, muscle, and heart, have emerged in both clinical trials and real-world post marketing settings. The 289th European NeuroMuscular Centre (ENMC) workshop convened 38 participants from patient advocacy groups, industry, and preclinical and clinical research groups to collaboratively review these toxicities, their underlying mechanisms, and potential mitigation and monitoring strategies. Discussions addressed the clinical spectrum and biological drivers of these events, the respective roles of innate and adaptive immunity, the contribution of specific vector characteristics as well as of the specific disease and recipient. The application of risk stratification and immunosuppressive regimens for prevention, monitoring, and management were considered. Emerging toxicities, including capillary leak syndrome, endothelial and dorsal root ganglia injuries, were reviewed alongside corresponding preclinical data from non-human primates. Participants agreed on the need to harmonize standard operating procedures, clinical guidelines, and data-sharing practices, and endorsed collaborative initiatives to proactively address critical gaps and unresolved key questions through a patient-centered framework.

Adaptive immune response

Current pediatric roles in child abuse and neglect.

Many pediatricians are being called on to undertake expanded roles in the field of child abuse and neglect, whether as a practicing pediatrician, a hospital-based child abuse consultant, or as a child protection team pediatrician. The practicing pediatrician must consider the diagnosis of child abuse and neglect, confirm the diagnosis, report all suspected cases to child protective services, hospitalize any abused child who needs protection, and provide preventive services. The hospital-based child abuse consultant should provide consultation to primary physicians, report seriously injured cases for the primary physician or surgeon, provide expert medical testimony on difficult to prove cases, teach house staff and medical students about child abuse and neglect, and improve treatment services for abused children who are hospitalized. The child protection team pediatrician will usually become involved in the broader problem of improving team decision making and the interagency system that deals with child abuse and neglect.

Child

The ward round.

Explore the source record for details and available documents.

Consumer Behavior

Doctor patient relationships and the older patient.

The authority of physicians in patient encounters is posited by the sicrole and the professional-client models in sociology. Yet, public challenges to physician authority are currently occurring, as shown by self care movements, and by some empirical data. Whether the elderly share in such challenges is, however, an unresolved question. Will longer experience with health services produce skepticism about medicine's claims, or conversely will the aged's probable more authoritarina upbringing and concern about failing health produce acceptance of physician authority? Data from an interview survey of 640 randomly sampled persons in a midwestern state, reveal that the 153 respondents aged 60 and over are more likely to accept physician authority, both in terms of attitudes and behaviors, than are the younger groups. Moreover, the findings show that the level of older persons' challenging attitudes is affected by their health knowledge and general views on authority, while their challenging behaviors are influenced by belief in right to medical information and an experience factor. Implications of the findings for appropriate modes of doctor-patient relationships for the elderly are discussed.

Adult