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Current and Emerging Therapies for Lysosomal Storage Disorders.
Drugs
January 2025
Lysosomal Storage Disorders Unit, Royal Free London NHS Foundation Trust, University College London, London, NW3 2QG, UK.
Lysosomal storage disorders (LSDs) are rare inherited metabolic disorders characterized by defects in the function of specific enzymes responsible for breaking down substrates within cellular organelles (lysosomes) essential for the processing of macromolecules. Undigested substrate accumulates within lysosomes, leading to cellular dysfunction, tissue damage, and clinical manifestations. Clinical features vary depending on the degree and type of enzyme deficiency, the type and extent of substrate accumulated, and the tissues affected.
View Article and Find Full Text PDFState-of-the-Art Liver Cancer Organoids: Modeling Cancer Stem Cell Heterogeneity for Personalized Treatment.
BioDrugs
January 2025
Orsay-Vallée Campus, Paris-Saclay University, Gif-sur-Yvette, France.
Liver cancer poses a global health challenge with limited therapeutic options. Notably, the limited success of current therapies in patients with primary liver cancers (PLCs) may be attributed to the high heterogeneity of both hepatocellular carcinoma (HCCs) and intrahepatic cholangiocarcinoma (iCCAs). This heterogeneity evolves over time as tumor-initiating stem cells, or cancer stem cells (CSCs), undergo (epi)genetic alterations or encounter microenvironmental changes within the tumor microenvironment.
View Article and Find Full Text PDFMuscle Loss During First-Line Chemotherapy Impairs Survival in Advanced Pancreatic Cancer Despite Adapted Physical Activity.
J Cachexia Sarcopenia Muscle
January 2025
Department of Medical Oncology, Lille University Hospital, Lille, France.
Background: Advanced pancreatic ductal adenocarcinoma (aPDAC) is often accompanied by significant muscle mass loss, contributing to poor prognosis. SarcAPACaP, an ancillary study of the GERCOR-APACaP phase III trial, evaluated the role of adapted physical activity (APA) in aPDAC Western patients receiving first-line chemotherapy. The study aimed to assess (1) the potential impact of computed tomography (CT)-quantified muscle mass before and during treatments on health-related quality of life (HRQoL) and overall survival (OS) and (2) the role of APA in mitigating muscle mass loss.
View Article and Find Full Text PDFIndependent macroscopic chemical mappings of cultural heritage materials with reflectance imaging spectroscopy: case study of a 16 century Aztec manuscript.
Anal Methods
November 2017
Centre de Recherche sur la Conservation (CRC), MNHN, Sorbonne-Universités CNRS, MCC, USR 3224, CP21, 36 rue Geoffroy Saint Hilaire, 75005 Paris, France.
Reflectance spectral imaging is a powerful tool for the non-invasive study of cultural heritage objects. Particular visible to short wave infrared (400-2500 nm) spectral features are linked to compositional information. Spectral images can hence be used to generate useful chemical maps.
View Article and Find Full Text PDFPanic disorder during pregnancy and the first three years after delivery: a systematic review.
BMC Pregnancy Childbirth
January 2025
Department of Psychiatry & Psychotherapy, Faculty of Medicine, Carl Gustav Carus University Hospital, Technische Universität Dresden, Dresden, Germany.
Background: Panic disorder (PD) is highly prevalent during the peripartum period. The aim of this systematic review was to summarize evidence on risk factors and course patterns of peripartum PD as well as maternal, infant or dyadic outcomes during the first three years after delivery.
Methods: A literature search was conducted according to PRISMA guidelines.
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