Lysosomal diseases (LDs) are a group of rare inherited disorders belonging to inborn metabolism errors. LDs are characterized by the excessive storage of undegraded substrates, most often due to the enzymatic deficiency resulting from disease-causing gene variants. LDs lead to dysregulated cellular pathways and imbalanced molecular homeostasis and can affect multiple organs and tissues. Despite being rare, LDs account for a significant incidence when considered collectively. Due to complex molecular and genetic fingerprints, considerable challenges in LD management must be overcome. Diagnosis can be significantly delayed due to the broad and nonspecific clinical manifestations and the lack of specific biomarkers. Available treatments fail to fully stop the disease progression and can alter the disease's typical phenotypes with novel manifestations. Therefore, a paradigm shift is crucial to better understand LDs and provide actionable insights. Herein, we comprehensively review the literature to demonstrate that multi-omics approaches are promising for pathophysiology elucidation, biomarker discovery, and precision therapy in LDs. We recommend adopting longitudinal study designs integrated with a multi-omics-empowered framework to facilitate mechanistic delineation, biomarker discovery, and treatment development. Relevant approaches exploring the association between LDs and common neurodegenerative disorders are also discussed, paving a potential path for improved therapeutic development and ultimately improving the patient's quality of life.

Purpose: This study aimed to examine current global practices in regenerative therapy (RT) for erectile dysfunction (ED) and to establish expert recommendations for its use, addressing the current lack of solid evidence and standardized guidelines. Materials and Methods: A 39-question survey was developed by senior Global Andrology Forum (GAF) experts to comprehensively cover clinical aspects of RT. This was distributed globally via a secure online Google Form to ED specialists through the GAF website, international professional societies, and social media, the responses were analyzed and presented for frequencies as percentages. Consensus on expert recommendations for RT use was achieved using the Delphi method. Results: Out of 479 respondents from 62 countries, a third reported using RT for ED. The most popular treatment was low-intensity shock wave therapy (54.6%), followed by platelet-rich plasma (24.5%) and their combination (14.7%), with stem cell therapy being the least used (3.7%). The primary indication for RT was the refractory or adverse effects of PDE5 inhibitors, with the best effectiveness reported in middle-aged and mild-to-moderate ED patients. Respondents were confident about its overall safety, with a significant number expressing interest in RT’s future use, despite pending guidelines support. Conclusions This inaugural global survey reveals a growing use of RT in ED treatment, showcasing its diverse clinical applications and potential for future widespread adoption. However, the lack of comprehensive evidence and clear guidelines requires further research to standardize RT practices in ED treatment.

Purpose: This study aimed to examine current global practices in regenerative therapy (RT) for erectile dysfunction (ED) and to establish expert recommendations for its use, addressing the current lack of solid evidence and standardized guidelines. Materials and Methods: A 39-question survey was developed by senior Global Andrology Forum (GAF) experts to comprehensively cover clinical aspects of RT. This was distributed globally via a secure online Google Form to ED specialists through the GAF website, international professional societies, and social media, the responses were analyzed and presented for frequencies as percentages. Consensus on expert recommendations for RT use was achieved using the Delphi method. Results: Out of 479 respondents from 62 countries, a third reported using RT for ED. The most popular treatment was low-intensity shock wave therapy (54.6%), followed by platelet-rich plasma (24.5%) and their combination (14.7%), with stem cell therapy being the least used (3.7%). The primary indication for RT was the refractory or adverse effects of PDE5 inhibitors, with the best effectiveness reported in middle-aged and mild-to-moderate ED patients. Respondents were confident about its overall safety, with a significant number expressing interest in RT’s future use, despite pending guidelines support. Conclusions This inaugural global survey reveals a growing use of RT in ED treatment, showcasing its diverse clinical applications and potential for future widespread adoption. However, the lack of comprehensive evidence and clear guidelines requires further research to standardize RT practices in ED treatment.

Purpose: This study aimed to examine current global practices in regenerative therapy (RT) for erectile dysfunction (ED) and to establish expert recommendations for its use, addressing the current lack of solid evidence and standardized guidelines. Materials and Methods: A 39-question survey was developed by senior Global Andrology Forum (GAF) experts to comprehensively cover clinical aspects of RT. This was distributed globally via a secure online Google Form to ED specialists through the GAF website, international professional societies, and social media, the responses were analyzed and presented for frequencies as percentages. Consensus on expert recommendations for RT use was achieved using the Delphi method. Results: Out of 479 respondents from 62 countries, a third reported using RT for ED. The most popular treatment was low-intensity shock wave therapy (54.6%), followed by platelet-rich plasma (24.5%) and their combination (14.7%), with stem cell therapy being the least used (3.7%). The primary indication for RT was the refractory or adverse effects of PDE5 inhibitors, with the best effectiveness reported in middle-aged and mild-to-moderate ED patients. Respondents were confident about its overall safety, with a significant number expressing interest in RT’s future use, despite pending guidelines support. Conclusions This inaugural global survey reveals a growing use of RT in ED treatment, showcasing its diverse clinical applications and potential for future widespread adoption. However, the lack of comprehensive evidence and clear guidelines requires further research to standardize RT practices in ED treatment.

Purpose: This study aimed to examine current global practices in regenerative therapy (RT) for erectile dysfunction (ED) and to establish expert recommendations for its use, addressing the current lack of solid evidence and standardized guidelines. Materials and Methods: A 39-question survey was developed by senior Global Andrology Forum (GAF) experts to comprehensively cover clinical aspects of RT. This was distributed globally via a secure online Google Form to ED specialists through the GAF website, international professional societies, and social media, the responses were analyzed and presented for frequencies as percentages. Consensus on expert recommendations for RT use was achieved using the Delphi method. Results: Out of 479 respondents from 62 countries, a third reported using RT for ED. The most popular treatment was low-intensity shock wave therapy (54.6%), followed by platelet-rich plasma (24.5%) and their combination (14.7%), with stem cell therapy being the least used (3.7%). The primary indication for RT was the refractory or adverse effects of PDE5 inhibitors, with the best effectiveness reported in middle-aged and mild-to-moderate ED patients. Respondents were confident about its overall safety, with a significant number expressing interest in RT’s future use, despite pending guidelines support. Conclusions This inaugural global survey reveals a growing use of RT in ED treatment, showcasing its diverse clinical applications and potential for future widespread adoption. However, the lack of comprehensive evidence and clear guidelines requires further research to standardize RT practices in ED treatment.

Purpose: This study aimed to examine current global practices in regenerative therapy (RT) for erectile dysfunction (ED) and to establish expert recommendations for its use, addressing the current lack of solid evidence and standardized guidelines. Materials and Methods: A 39-question survey was developed by senior Global Andrology Forum (GAF) experts to comprehensively cover clinical aspects of RT. This was distributed globally via a secure online Google Form to ED specialists through the GAF website, international professional societies, and social media, the responses were analyzed and presented for frequencies as percentages. Consensus on expert recommendations for RT use was achieved using the Delphi method. Results: Out of 479 respondents from 62 countries, a third reported using RT for ED. The most popular treatment was low-intensity shock wave therapy (54.6%), followed by platelet-rich plasma (24.5%) and their combination (14.7%), with stem cell therapy being the least used (3.7%). The primary indication for RT was the refractory or adverse effects of PDE5 inhibitors, with the best effectiveness reported in middle-aged and mild-to-moderate ED patients. Respondents were confident about its overall safety, with a significant number expressing interest in RT’s future use, despite pending guidelines support. Conclusions This inaugural global survey reveals a growing use of RT in ED treatment, showcasing its diverse clinical applications and potential for future widespread adoption. However, the lack of comprehensive evidence and clear guidelines requires further research to standardize RT practices in ED treatment.

Globally, providing evidence on the economic burden of chronic obstructive pulmonary disease (COPD) is becoming essential as it assists the health authorities to efficiently allocate resources. This study aimed to summarize the literature on economic burden evidence for COPD from 1990 to 2019. This study examined the economic burden of COPD through a systematic review of studies from 1990 to 2019. A search was done in online databases, including Web of Science, PubMed/Medline, Scopus, and the Cochrane Library. After screening 12,734 studies, 43 articles that met the inclusion criteria were identified. General study information and data on direct, indirect, and intangible costs were extracted and converted to 2018 international dollars (Int$). Findings revealed that the total direct costs ranged from Int$ 52.08 (India) to Int$ 13,776.33 (Canada) across 16 studies, with drug costs rannging from Int$ 70.07 (Vietnam) to Int$ 8,706.9 (China) in 11 studies. Eight studies explored indirect costs, while one highlighted caregivers’ direct costs at approximately Int$ 1,207.8 (Greece). This study underscores the limited research on COPD caregivers’ economic burdens, particularly in developing countries, emphasizing the importance of increased research support, particularly in high-resource settings. This study provides information about the demographics and economic burden of COPD from 1990 to 2019. More strategies to reduce the frequency of hospital admissions and acute care services should be implemented to improve the quality of COPD patients’ lives and reduce the disease’s rising economic burden.

The spread of tuberculosis (TB), especially multidrug-resistant TB and extensively drug-resistant TB, has strongly motivated the research and development of new anti-TB drugs. New strategies to facilitate drug combinations, including pharmacokinetics-guided dose optimization and toxicology studies of first- and second-line anti-TB drugs have also been introduced and recommended. Liquid chromatography-mass spectrometry (LC-MS) has arguably become the gold standard in the analysis of both endo- and exo-genous compounds. This technique has been applied successfully not only for therapeutic drug monitoring (TDM) but also for pharmacometabolomics analysis. TDM improves the effectiveness of treatment, reduces adverse drug reactions, and the likelihood of drug resistance development in TB patients by determining dosage regimens that produce concentrations within the therapeutic target window. Based on TDM, the dose would be optimized individually to achieve favorable outcomes. Pharmacometabolomics is essential in generating and validating hypotheses regarding the metabolism of anti-TB drugs, aiding in the discovery of potential biomarkers for TB diagnostics, treatment monitoring, and outcome evaluation. This article highlighted the current progresses in TDM of anti-TB drugs based on LC-MS bioassay in the last two decades. Besides, we discussed the advantages and disadvantages of this technique in practical use. The pressing need for non-invasive sampling approaches and stability studies of anti-TB drugs was highlighted. Lastly, we provided perspectives on the prospects of combining LC-MS-based TDM and pharmacometabolomics with other advanced strategies (pharmacometrics, drug and vaccine developments, machine learning/artificial intelligence, among others) to encapsulate in an all-inclusive approach to improve treatment outcomes of TB patients.

Objective: The risk of transmission of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) from schoolchildren to their household and the protective effects of vaccination in these settings remain poorly understood. We assessed the transmission dynamics of schoolchildren with SARS-CoV-2 within their households and the protective effects of coronavirus disease (COVID-19) vaccination among household members in Viet Nam. Methods: We estimated the attack rate, vaccine effectiveness and adjusted risk ratio (aRR) of factors associated with SARS-CoV-2 transmission to household contacts of children confirmed to have COVID-19 who attended three schools in Ha Nam, Phu Tho and Thanh Hoa provinces between September and December 2021 using multivariable regression with household-level random effects. Results: This retrospective cohort study included 157 children infected with SARS-CoV-2 and their 540 household contacts. The attack rate among household contacts was 24.6% (133/540). Overall, vaccine effectiveness among household contacts was 39% (95% confidence interval [CI]: -1 to -63), higher among males than females and higher in adults aged >40 years. COVID-19 transmission was greater among female household contacts compared with males (aRR: 1.35, 95% CI: 0.94 to 1.95), although not statistically significant, and highest among those aged 19–39 years (aRR: 2.51, 95% CI: 1.50 to 4.21). Fully vaccinated household contacts had significantly lower infection risk (aRR: 0.46, 95% CI: 0.26 to 0.84). Discussion: We found substantial onward transmission of SARS-CoV-2 from schoolchildren to household members, and older people were more likely to be protected by vaccination. We recommend that schoolchildren and all household members living with schoolchildren receive at least two doses of a COVID-19 vaccine. Recognizing the role of schoolchildren in the onward transmission of COVID-19 is an important lesson learned by Viet Nam that can help not only in managing other outbreaks but also in protecting schoolchildren by predicting the progress of the outbreak and preparing for a timely response.

Background: s/Aims: Pancreaticoduodenectomy (PD) is the only radical treatment for periampullary malignancies. Superior mesenteric artery (SMA) first approach combined with total meso-pancreas (MP) excision was conducted to improve the oncological results.There has not been any previous research of a technique that combines the SMA first approach and total MP excision with a detailed description of the MP macroscopical shape. Methods: We prospectively assessed 77 patients with periampullary malignancies between October 2020 and March 2022 (18 months). All patients had undergone PD with SMA first approach combined total MP excision. The perioperative indications, clinical data, intra-operative index, R0 resection rate of postoperative pathological specimens (especially mesopancreatic margin), postoperative complications, and follow-up results were evaluated. Results: The median operative time was 289.6 min (178−540 min), the median intraoperative blood loss was 209 mL (30−1,600 mL).Microscopically, there were 19 (24.7%) cases with metastatic MP, and five cases (6.5%) with R1-resection of the MP. The number of lymph nodes (LNs) harvested and metastatic LNs were 27.2 (maximum was 74) and 1.8 (maximum was 16), respectively. Some (46.8%) patients had pancreatic fistula, but mostly in grade A, with 7 patients (9.1%) who required re-operations. Some 18.2% of cases developed postoperative refractory diarrhea. The rate of in-hospital mortality was 1.3%. Conclusions The PD with SMA first approach combined TMpE for periampullary malignancies was effective in achieving superior oncological statistics (rate of MP R0-resection and number of total resected LNs) with non-inferior short-term outcomes. It is necessary to evaluate survival outcomes with long-term follow-up.