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Two-year follow-up after drug desensitization in mucopolysaccharidosis.
Orphanet J Rare Dis
December 2024
Post Graduate School in Allergology and Internal Medicine "Guido Baccelli", Department of Precision and Regenerative Medicine and Ionian Area-(DiMePRe-J), School of Medicine, Aldo Moro University of Bari, Bari, 70124, Italy.
Background: Mucopolysaccharidosis (MPS) type 1 S and type 2 are rare lysosomal storage disorders characterized by impaired enzyme production, resulting in glycosaminoglycans accumulation within lysosomes. Enzyme Replacement Therapy (ERT) with laronidase and idursulfase are first line treatments, respectively. However, infusion-related hypersensitivity reactions (HR) may lead to ERT discontinuation.
View Article and Find Full Text PDFLetter to the editor: "Levacetylleucine a game changer for Niemann pick disease type-C".
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Karachi Medical and Dental College, Karachi, Sindh, Pakistan.
LAMTOR1 decreased exosomal PD-L1 to enhance immunotherapy efficacy in non-small cell lung cancer.
Mol Cancer
September 2024
Department of Pulmonary and Critical Care Medicine, Shengjing Hospital of China Medical University, Shenyang, 110136, China.
Great progress has been made in utilizing immune checkpoint blockade (ICB) for the treatment of non-small-cell lung cancer (NSCLC). Therapies targeting programmed cell death protein 1 (PD-1) and its ligand PD-L1, expressed on tumor cells, have demonstrated potential in improving patient survival rates. An unresolved issue involves immune suppression induced by exosomal PD-L1 within the tumor microenvironment (TME), particularly regarding CD8 T cells.
View Article and Find Full Text PDFResponse to Comments on "Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe Mice".
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Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area, Sanofi, Cambridge, Massachusetts.
We thank Dr. Weimer and her colleagues for their comments related to our recent work (Anding et al., 2023) and are grateful for the opportunity to further discuss the importance of efficient lysosomal targeting of enzyme-replacement therapies (ERT) for the treatment of Pompe disease.
View Article and Find Full Text PDFUnraveling Phenotypic Variability in Action Myoclonus with Renal Failure with SCARB2 Mutation in Siblings.
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Comprehensive Care Centre for Movement Disorders (CCCMD), Department of Neurology, Sree Chitra Tirunal Institute for Medical Sciences and Technology, Thiruvananthapuram, India.
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