Results 161 to 170 of about 460,260 (336)

Plasmatic and urinary glycosaminoglycans characterization in mucopolysaccharidosis II patient treated with enzyme-replacement therapy with idursulfase [PDF]

, 2012
Plasmatic and urinary glycosaminoglycans characterization in mucopolysaccharidosis II patient treated with enzyme-replacement therapy with ...
VOLPI, Nicola
core  

Profile of asfotase alfa in the treatment of hypophosphatasia: design, development, and place in therapy

Drug Design, Development and Therapy, 2018
Sasigarn A Bowden,1 Brian L Foster2 1Division of Endocrinology, Department of Pediatrics, Nationwide Children’s Hospital/The Ohio State University College of Medicine, Columbus, OH 43205, USA; 2Division of Biosciences, College of Dentistry, The ...
Bowden SA, Foster BL
doaj  

Electrostatically‐Stabilized PEG‐Free Lipid Nanoparticles for Systemic Nucleic Acid Delivery

Advanced Functional Materials, EarlyView.
This work describes non‐PEGylated layered lipid nanoparticles (nonPEG LLNPs), a gene delivery platform that improves upon standard PEGylated LNPs via electrostatic adsorption of charged polymers. nonPEG LLNPs maintain colloidal stability under biological stresses, enhance circulation time, and mitigate accelerated blood clearance and hepatic ...
Namita Nabar, Deeksha Kumaresh, Eduardo Nombera Bueno, Mae Pryor, Paula T. Hammond   +4 more
wiley   +1 more source

UBE3A Enzyme Replacement Therapy for Angelman Syndrome [PDF]

Angelman Syndrome (AS) is a rare neurodevelopmental disorder caused by mutations in the UBE3A gene, resulting in a lack of UBE3A protein within neuronal cells due to the silencing of the paternal UBE3A gene.
Correa, Delihla, Bailus, Barbara, Dr., Stoub, Rachel   +2 more
core  

A 3D Human Neuron‐on‐Chip Platform to Monitor Neuronal Injury Responses

Advanced Healthcare Materials, EarlyView.
This study presents a novel 3D Neuron‐on‐Chip model that can maintain human PSC‐derived excitatory prefrontal cortex neurons in 3D hydrogels and can be used to monitor neuronal injury responses over time. Results show injury‐induced acute neuronal excitotoxicity, declining neuronal connectivity, and the activation of a neurodegenerative, SASP‐like ...
Ruiping Tang, Charles‐Francois Latchoumane, Avi Chopra, Md Marzan Sarkar, Chunki Kim, Nathan Gonsalves, Hsueh‐Fu Wu, John C. Sentmanat, Alan Liu, Isha Mhatre‐Winters, Aditya Mishra, Andrei G. Fedorov, Jay M. Patel, Nadja Zeltner, Steven L. Stice, Jason R. Richardson, Lohitash Karumbaiah   +16 more
wiley   +1 more source

Clinical course and management of fabry disease in three patients before the introduction of enzyme replacement therapy

Zdravniški Vestnik, 2006
Background: Fabry disease (FD) is an X-linked inherited disorder of glycosphingolipids metabolism. Clinical manifestations, the course and the cause of death are markedly different among the patients. Before the use of enzyme replacement therapy patients
Franc Verovnik
doaj  

Chamber‐Specific Decellularized Extracellular Matrices Differentially Modulate Cardiomyocyte Subtypes to Drive Engineered Heart Tissue Development and Function

Advanced Healthcare Materials, EarlyView.
Chamber‐specific decellularized extracellular matrices (ECMs) were developed, preserving native proteomic profiles of ventricular and atrial myocardium. These innate biochemical cues differentially modulate cardiomyocyte subtypes to drive engineered heart tissue development and function, highlighting the importance of incorporating regional ECM cues in
Dong Gyu Hwang, Myungji Kim, Hwanyong Choi, Jinseon Park, Minji Kim, Donghwan Kim, Bo Ram Lee, Jinah Jang   +7 more
wiley   +1 more source

Enzyme replacement therapy in India

Journal of Postgraduate Medicine, 2018
Muranjan, M, Karande, S
openaire   +2 more sources

Microfluidic Nano‐Assembly of Red‐Blood‐Cell (RBC) Lipids and Components for Engineering Extracellular Vesicles

Advanced Healthcare Materials, EarlyView.
Engineered red blood cell‐derived extracellular vesicles (eRBCEVs) are synthesized via controlled microfluidic assembly from native RBC lipids, enabling tunable encapsulation of proteins, nucleic acids, nanoparticles, and viral vectors. The platform demonstrates reproducible nanoscale architecture, preserved membrane composition, and functional cargo ...
Chiranth K. Nagaraj, Xilal Y. Rima, Kim T. Nguyen, Courtney Culkins, Nana Boateng, Jacob Doon‐Ralls, Alejandro Bresolin, Xin Huang, Hong Li, Vahedi Amid, Ajeet Pal Singh, Dharti Shantaram, Anastasiia Amari, Mangesh D. Hade, Nicholas R. Merriam, Zachary D. Schultz, Scott Q. Harper, Nizar Y. Saad, Setty M. Magaña, Willa A. Hsueh, Rachel E. Kopec, Blaise R. Kimmel, Andre F. Palmer, Eduardo Reátegui   +23 more
wiley   +1 more source

Inflammation Unchecked: Concurrent Kawasaki Disease and Stevens‐Johnson Syndrome in an 18‐Month‐Old Child

Arthritis Care &Research, EarlyView.
Catherine Deffendall, Jodi Backalar, Elizabeth Acevedo Zapata, Adriana Della Porta, Lorin Bibb, Tyler Reese, Charlotte M. Brown   +6 more
wiley   +1 more source