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Good Practices for Health Technology Assessment Guideline Development: A Report of the Health Technology Assessment International, HTAsiaLink, and ISPOR Special Task Force.
Value Health
October 2024
Health Intervention and Technology Assessment Program (HITAP), Ministry of Public Health, Nonthaburi, Thailand; Saw Swee Hock School of Public Health, National University of Singapore, Singapore.
Objectives: Health technology assessment (HTA) guidelines are intended to support successful implementation of HTA by enhancing consistency and transparency in concepts, methods, process, and use, thereby enhancing the legitimacy of the decision-making process. This report lays out good practices and practical recommendations for developing or updating HTA guidelines to ensure successful implementation.
Methods: The task force was established in 2022 and comprised experts and academics from various geographical regions, each with substantial experience in developing HTA guidelines for national health policymaking.
Practical Recommendations for the Diagnosis and Management of Lysosomal Acid Lipase Deficiency with a Focus on Wolman Disease.
Nutrients
December 2024
Pediatric Hepatology and Liver Transplant Unit, Department of Pediatrics, ERN Rare Liver ERN TransplantChild, Vall d'Hebron Barcelona Hospital Campus, Universitat Autònoma de Barcelona, 08193 Barcelona, Spain.
Lysosomal acid lipase deficiency (LAL-D) is an ultra-rare lysosomal storage disease with two distinct phenotypes, an infantile-onset form (formerly Wolman disease) and a later-onset form (formerly cholesteryl ester storage disease). The objective of this narrative review is to examine the most important aspects of the diagnosis and treatment of LAL-D and to provide practical expert recommendations. The infantile-onset form occurs in the first weeks of life and is characterized by malnourishment and failure to thrive due to gastrointestinal impairment (vomiting, diarrhea, malabsorption), as well as systemic inflammation, hepatosplenomegaly, and adrenal calcifications.
View Article and Find Full Text PDFEmbryoid Body Test: A Simple and Reliable Alternative Developmental Toxicity Test.
Int J Mol Sci
December 2024
Laboratory of Veterinary Biochemistry and Molecular Biology, College of Veterinary Medicine, Chungbuk National University, Cheongju 28644, Republic of Korea.
The increasing emphasis on animal welfare and ethics, as well as the considerable time and cost involved with animal testing, have prompted the replacement of many aspects of animal testing with alternative methods. In the area of developmental toxicity, the embryonic stem cell test (EST) has played a significant role. The EST evaluates toxicity using mouse embryonic stem cells and somatic cells and observes the changes in heartbeat after cardiac differentiation.
View Article and Find Full Text PDFWomen with IBD Show Higher Psychophysiological Burden in Comparison to Men with IBD.
J Clin Med
December 2024
Goethe University Frankfurt, Frankfurt University Hospital, Medical Clinic 1, 60596 Frankfurt am Main, Germany.
Inflammatory bowel disease (IBD) remains an incurable illness. Patients with IBD show gender-specific differences in various aspects of the disease. There is still uncertainty about the causality of the differences.
View Article and Find Full Text PDFEffectiveness of Telemedicine in Inflammatory Bowel Disease in Russia: TIGE-Rus (Telemonitoring for IBD Goodness Examination in Russia) Study Protocol of a Randomized Controlled Trial.
J Clin Med
December 2024
Research and Practical Clinical Center for Diagnostics and Telemedicine Technologies of the Moscow Health Care Department, Moscow 127051, Russia.
: Inflammatory bowel diseases (IBD), associated with a significant burden on patients' lives, are becoming increasingly common. Patients with IBD need continuous treatment and lifelong monitoring, which could be achieved by telemonitoring. Telemonitoring has been shown to be effective in improving outcomes for patients with IBD, and can provide a more convenient and accessible way for patients to receive care.
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