Diagnosis and follow-up of the first case of Gaucher disease under enzyme replacement therapy in Senegal. [PDF]
Keita M +7 more
europepmc +1 more source
ABSTRACT The ascent of novel alternative methods in drug development spotlights the dual needs for improved biological fidelity to in vivo, along with reproducibility, especially in regulatory applications. The need for pre‐clinical models of patient‐derived endometriosis lesions motivates the development of a vascularizable, completely synthetic ...
Lauren Pruett +7 more
wiley +1 more source
Enzyme replacement therapy during pregnancy and breastfeeding in late-onset Pompe disease. [PDF]
Bachmann M +7 more
europepmc +1 more source
Multidimensional Cellular Micro‐Compartments to Model Invasive Lobular Carcinoma Dormancy
Invasive lobular carcinoma (ILC) is an understudied subtype of breast cancer that is susceptible to late recurrences. In this study, micro‐compartmentalization techniques spanning multiple dimensions, including 2D, pseudo‐3D, and 3D, are integrated to uncover the mechanisms underlying ILC dormancy, revealing the central role of p27Kip1.
Xilal Y. Rima +15 more
wiley +1 more source
Recombinant ADAMTS13: An Enzyme Replacement Therapy for the Management of Congenital Thrombotic Thrombocytopenic Purpura. [PDF]
Robichaux TP +4 more
europepmc +1 more source
Copper–Collagen Interactions Regulate the Mechanical and Invasive Properties of Tumor Spheroids
Ionic copper is investigated as a modulator of tumor spheroid growth and invasiveness in a 3D microfluidic glioblastoma model. Copper exposure alters metabolic activity, cytoskeletal organization, protrusion dynamics, and EMT‐related phenotypes in a dose‐ and timing‐dependent manner, revealing how copper bioavailability shapes tumor aggressiveness ...
Paula Guerrero‐López +5 more
wiley +1 more source
Increased intervals in enzyme replacement therapy for stable type 1 Gaucher disease: A non-inferiority sequential trial emulation. [PDF]
Beydon M +31 more
europepmc +1 more source
Aptamer‐Based Delivery Systems for VEGF and NGF Modulation in Ocular Therapies
Aptamer‐based delivery systems targeting NGF and VEGF hold significant potential for precise and sustained treatment of complex diseases affecting both the anterior and posterior segments of the eye. This review critically summarizes current strategies for NGF and VEGF delivery as well as VEGF sequestration, providing a clinical perspective on how ...
Nadine Best +5 more
wiley +1 more source
Management of life-threatening anaphylaxis to enzyme replacement therapy in an infant with Pompe disease: a case report and literature review. [PDF]
Zhou J +6 more
europepmc +1 more source
Plasmatic and urinary glycosaminoglycans characterization in mucopolysaccharidosis II patient treated with enzyme-replacement therapy with ...
VOLPI, Nicola
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