Gene therapy-based strategies for spinal muscular atrophy-an Asia-Pacific perspective.

Onasemnogene abeparvovec has been life-changing for children with spinal muscular atrophy (SMA), signifying the potential and progress occurring in gene- and cell-based therapies for rare genetic diseases. Hence, it is important that clinicians gain knowledge and understanding in gene therapy-based treatment strategies for SMA. In this review, we describe the development and translation of onasemnogene abeparvovec from clinical trials to healthcare practice and share knowledge on the facilitators and barriers to implementation. Rapid and accurate SMA diagnosis, awareness, and education to safely deliver gene therapy to eligible patients and access to expertise in multidisciplinary management for neuromuscular disorders are crucial for health system readiness. Early engagement and intersectoral collaboration are required to surmount complex logistical processes and develop policy, governance, and accountability. The collection and utilisation of real-world evidence are also an important part of clinical stewardship, informing ongoing improvements to care delivery and access. Additionally, a research-enabled clinical ecosystem can expand scientific knowledge and discovery to optimise future therapies and magnify health impacts. Important ethical, equity, economic, and sustainability issues are evident, for which we must connect globally.

The association of online search interest with polio cases and vaccine coverage: an infodemiological and ecological study.

Achievement of universal eradication of paralytic poliomyelitis has remained a challenge. Despite the general decline in cases, multiple outbreaks attributed to poor vaccination still occur. Noncompliance from vaccination can be improved through education on various media platforms. In the internet age, online health-seeking behavior plays a significant role in this regard. Hence, our study investigated the association between global online search interest in polio with the number of polio cases and vaccination coverage. This infodemiological and ecological study utilized Google Trends' search volume index (SVI) for "polio" and the World Health Organization data on the number of polio cases (PC) and vaccine coverage rate (VCR) per country between 2006 and 2019. Associations between SVI for "polio" with PC and with VCR were evaluated. From the years 2006 to 2019, the global inquiry for this term was highest (i.e., SVI at 100) last October 2018. There was a direct correlation between the SVI for "polio" and PC while there was an inverse relationship between SVI and VCR per country per year. Both relationships have weak to moderate strength of associations. Based on our models, a one-unit increase in the SVI leads to a 3.8% increase in the number of polio cases. On the other hand, a one-unit increase in the SVI leads to a 0.01% decrease in the VCR.Conclusions: Dynamic changes in global SVIs for polio may reflect fluctuations in the number of polio cases and rates of vaccine coverage. Our study brings into light the largely untapped and potential use of online search behavior for polio to anticipate changes in PC and VCR in real-time. What is Known: •Parental vaccine hesitancy is a strong hindrance to the eradication of vaccine-preventable diseases. •The internet is a major source of information that modifies this attitude. What is New: •Internet health-seeking behavior can be measured using Google Trends' search volume index and can be used to correlate to certain aspects of public health determinants of a certain disease. •Google Trends' search volume index correlates with the number of polio cases/immunization rates, and this provides a basis for considering public health measures online.

Prophylactic intravenous ketorolac for prevention of intraventricular hemorrhage in preterm infants ? 32 weeks and < 1500 grams: A double-blind, randomized, placebo-controlled trial

@#<p style="text-align: justify;">Intraventricular hemorrhage (IVH) remains an important cause of morbidity and mortality in Very Low Birth Weight (VLBW) infants. Since 2004, Indomethacin, which is effective in preventing IVH, has been removed from the Philippine market. Ketorolac is a nonselective cyclooxygenase inhibitor which is structurally-related and of equal potency to Indomethacin.<br /><strong>OBJECTIVE:</strong> This study aims to determine if prophylactic ketorolac compared to placebo will decrease IVH and its associated morbidities among preterm neonates.<br /><strong>METHODS:</strong> We conducted a double-blind, randomized, placebo-controlled trial among neonates born in a tertiary government university hospital. Newborns with gestational age ?32 weeks and birth weight <strong>RESULTS:</strong> A total of 134 infants were included in this study. There was no difference in the proportion of infants who developed IVH between the ketorolac and placebo groups (46% vs. 45%). The mean serum creatinine levels were significantly higher in the ketorolac group (1.15 ± 0.69 vs 0.79 ±0.38; p=0.002). The rates of death, sepsis, necrotizing enterocolitis, bleeding, platelet counts of <50,000/mm3, mean urine output and the lengths of hospital stay were similar in the two groups.<br /><strong>CONCLUSION:</strong> Prophylactic intravenous ketorolac was ineffective in preventing IVH among preterm infants. Ketorolac cannot be recommended for the prevention of IVH.</p>