[Quercetin, a key bioactive component of Zuogui Pill, protects against Alzheimer's disease by regulating the PI3K/Akt signaling pathway].

Spinal muscular atrophy (SMA) is a progressive neuromuscular disorder characterized by motor decline, respiratory insufficiency and orthopedic complications. Novel disease-modifying therapies have improved life expectancy particularly with early treatment afforded by newborn screening programs. These therapies include (1) onasemnogene abeparvovec, a one-time gene replacement therapy delivered intravenously, (2) nusinersen, an antisense oligonucleotide delivered intrathecally at regular intervals, and (3) risdiplam, a small molecule splicing modifier drug delivered orally. In this new era, children with SMA and their families face a high burden of morbidity, medical care needs, and prognostic uncertainty. Decision-making is complex for people with SMA and their caregivers as well as practitioners, and heavily influenced by the local clinical setting and resource availability. Social determinants of health (e.g., education access and quality, health care and quality, neighborhood and built environment, social and community context, and economic stability as categorized by the Centers for Disease Control and Prevention (CDC)) influence how caregivers approach decision-making both as direct and indirect factors. The social determinants of health have significant implications for children living with medical complexity and life-limiting illnesses. Lower socioeconomic status groups are disproportionately impacted by work disruption and the financial burden of caring for a child with medical complexity, more affected by issues of stigma, and less likely to access advanced care resources. These social considerations are intricately related to the Four Principles of Western biomedical ethics including (1) beneficence - working in the best interest of the child, (2) nonmaleficence (i.e, refraining from or minimizing harm), (3) autonomy - self-determination, and (4) justice, including equity and distributive justice. Better appreciation of the ethical and social factors affecting the care experience of children with SMA can improve the ability of clinicians to deliver family-centered care, address unique challenges faced by families, and participate in decision-making conversations about the type of disease-modifying therapy to initiate. To our knowledge, no studies to date have examined the personal, psychosocial and systemic factors influencing the care experience of children and families with SMA following the advent of disease-modifying therapies. Using the Four Principles Bioethics framework and the CDC framework for social determinants of health, the aim of this present study was to explore the ethical issues and social determinants of health impacting the care experiences of caregivers for children with SMA receiving disease-modifying therapies within a publicly funded healthcare system.