Results 301 to 310 of about 532,411 (388)

Impact of enzyme replacement therapy on clinical manifestations in females with Fabry disease. [PDF]

Orphanet J Rare Dis
Lenders M, Nowak A, Cybulla M, Kaufeld J, Köhn AF, Muschol NM, Kurschat C, Brand E.   +7 more
europepmc   +1 more source

Advances in pancreatic enzyme replacement therapy for chronic pancreatitis

Ling-Ying Jiang, Miao Liu, Liang-Hao Hu
openalex   +1 more source

Decellularized Extracellular Matrix (dECM) in Tendon Regeneration: A Comprehensive Review

Advanced Healthcare Materials, EarlyView.
Decellularized Extracellular Matrix (dECM) offers a promising solution by replicating the native tendon microenvironment and promoting regeneration. This review highlights advances in the decellularization methods, as well as their integration with emerging technologies and translational progress in tendon tissue engineering.
Kumaresan Sakthiabirami, Yeonwoo Park, Seong Kyeong Jo, Min Hee Kim, Jae Hwang Song   +4 more
wiley   +1 more source

Assessing the Value for Money of Enzyme Replacement Therapy in Gaucher Disease Types 1 and 3b: Can Expanded Coverage Be Justified? [PDF]

Pharmacoecon Open
Rattanavipapong W, Anothaisintawee T, Isaranuwatchai W, Wattanasirichaigoon D, Tim-Aroon T, Wichajarn K, Sathienkijkanchai A, Charoenkwan P, Suphapeetiporn K, Traivaree C, Kuptanon C, Teerawattananon Y.   +11 more
europepmc   +1 more source

Strategies to Improve the Lipophilicity of Hydrophilic Macromolecular Drugs

Advanced Healthcare Materials, EarlyView.
Hydrophilic macromolecular drugs can be successfully lipidized by covalent attachment of lipids, by hydrophobic ion pairing with negatively or positively charged surfactants, and by dry or wet reverse micelle formation. Lipophilicity enhancement of hydrophilic macromolecules has several benefits including stability and bioavailability improvement ...
Sera Lindner, Stefan Keim, Soheil Haddadzadegan, Odile Fernandez Romero, Katrin Zöller, Gabriel Stern, Ilaria Cesi, Krum Kafedjiiski, Andreas Bernkop‐Schnürch   +8 more
wiley   +1 more source

Targeted nanoliposomes to improve enzyme replacement therapy of Fabry disease. [PDF]

Sci Adv
Tomsen-Melero J, Moltó-Abad M, Merlo-Mas J, Díaz-Riascos ZV, Cristóbal-Lecina E, Soldevila A, Altendorfer-Kroath T, Danino D, Ionita I, Pedersen JS, Snelling L, Clay H, Carreño A, Corchero JL, Pulido D, Casas J, Veciana J, Simó Schwartz Jr, Sala S, Font A, Birngruber T, Royo M, Córdoba A, Ventosa N, Abasolo I, González-Mira E.   +25 more
europepmc   +1 more source

Photosensitizing Lipid Nanoparticles for Ferroptosis‐Enhanced Photodynamic Cancer Therapy via GPX4 Silencing

Advanced Healthcare Materials, EarlyView.
Photosensitizing lipid nanoparticles (PLNPs) are engineered by incorporating cholesterol–PEG–pheophorbide a into MC3‐based LNPs and encapsulating GPX4‐targeting siRNA. Upon light activation, PLNPs generate reactive oxygen species (ROS) while silencing GPX4 to induce ferroptosis.
Ga‐Hyun Bae, Seungyong Shin, Joo Dong Park, Eun‐Young Koh, Seunghyo Ko, Jieun Han, Chun Gown Park, Dong‐Hyun Kim, Kun Na, Wooram Park   +9 more
wiley   +1 more source

Novel transferrin receptor-mediated enzyme replacement therapy efficiently treats myogenic and neurogenic aspects of Pompe disease in mice. [PDF]

Mol Ther Methods Clin Dev
George K, Riley R, Zhou S, Allen E, Smith L, Kistanova E, Kinton S, Guo L, Bangari D, Ismail A, Thummapudi J, Boumezbeur F, Selingue E, Tamarelle D, Capdevila C, Do TM, Lesuisse D, Leksa N, Sardi P, van der Flier A.   +19 more
europepmc   +1 more source

Advances in Bioprinting to Model Immune‐Mediated Skin Diseases

Advanced Healthcare Materials, EarlyView.
This review explores how 3D bioprinting drives innovation in developing in vitro skin models that mimic immune‐mediated diseases. It highlights current technologies, key applications in studying skin pathologies, and emerging challenges. The review points toward future opportunities for improving disease modeling and advancing therapeutic and cosmetic ...
Andrea Ulloa‐Fernández, Marica Markovic, Julia Fernández‐Pérez, Georg Stary, Aleksandr Ovsianikov   +4 more
wiley   +1 more source

Liver transplantation and portacaval shunt in genetic diseases [PDF]

, 1982
Starzl, TE
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