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GM1‐gangliosidosis (GM1) is a rare neurodegenerative disorder leading to early mortality and causing progressive decline of physical skills and cerebral functioning. No approved treatment for GM1 exists. In this study—the first to explore priorities of parents of subjects with pediatric onset forms of GM1—we address a crucial gap by characterizing symptoms most critical to caregivers of children with GM1 to treat. Our two‐part, mixed‐methods approach began with focus groups, followed by interviews with a distinct set of parents. Interviews included a prioritization activity that used best‐worst scaling. Quantitative data were analyzed descriptively. Qualitative data were analyzed using thematic analysis and rapid analysis process. Parents prioritized the symptoms they believed would increase their child's lifespan and improve their perceived quality of life (QoL); these symptoms focused on communicating wants/needs, preventing pain/discomfort, getting around and moving one's body, and enhancing eating/feeding. Although lifespan was highly valued, almost all parents would not desire a longer lifespan without acceptable child QoL. Parents indicated high caregiver burden and progressive reduction in QoL for children with GM1. This novel study of caregiver priorities identified important symptoms for endpoints' selection in patient‐focused drug development in the context of high disease impact and unmet treatment needs. [ABSTRACT FROM AUTHOR]

Background: The prevalence of deaths involving synthetic opioids has historically been lower in Texas than most U.S. states but more than quadrupled from January 2020 to January 2022. This paper explores the emergence of fentanyl in a drug supply where black tar heroin predominates, a factor considered protective against fentanyl adulteration, through the perspectives of people who use drugs (PWUD). Objectives: We describe experiences of unintentional exposure to fentanyl, illustrate how some people identify fentanyl in their supply, and present harm reduction strategies that PWUD use to avoid overdose. Methods: Thirty rapid assessment interviews were conducted in July 2021 at 2 mobile outreach sites of a harm reduction organization in Austin, Texas. The brief semistructured interviews were designed to assess participant fentanyl exposure experiences. Results: Participants were clients who reported using heroin or fentanyl in the past week and had lived in Texas for at least 6 months. Seventeen participants identified as male, 10 as female, and 3 as nonbinary. Half identified as white; other participants were Latinx (6), black (2), American Indian (1), and mixed race (6). Two-thirds were unhoused or in transitional housing. The drug supply in Texas has evolved; most participants reported that the heroin and other drugs they obtained contain fentanyl. Participants detected differences by observing changes in the physical characteristics of the drug, experiencing unexpected effects, and using fentanyl test strips. Many had been unintentionally exposed to fentanyl and expressed concerns about fentanyl’s presence. The presence of fentanyl had negative unintended consequences for participants, including adverse effects and developing a dependence on opioids. Conclusion: PWUD in Austin, Texas, report increasing prevalence of unintentional fentanyl exposure, despite the predominance of black tar heroin. Pharmacists can provide crucial supplies and education to safeguard the health of this vulnerable population. [ABSTRACT FROM AUTHOR]