Results 241 to 250 of about 460,260 (336)

Kinetic mechanism of ENPP1 ATPase: Implications for aberrant calcification disorders and enzyme replacement therapy. [PDF]

J Biol Chem
Michalchik MM, Potchernikov T, Lester ER, Braddock DT, Cao W, De La Cruz EM.   +5 more
europepmc   +1 more source

Cross‐Scale Hierarchical Targeted Delivery System Based on Small‐Scale Magnetic Robots

Advanced Robotics Research, EarlyView.
This article reviews a cross‐scale hierarchical targeted delivery system that integrates magnetic continuum robots and magnetic microrobots. By combining rapid long‐range navigation with precise microscale targeting, the system overcomes key limitations of single‐scale approaches.
Junjian Zhou, Zhiqiang Chen, Yuanbiao Ma, Zhiqiang Tang, Qianqian Wang   +4 more
wiley   +1 more source

Home Enzyme Replacement Therapy in Gaucher Disease: A Review. [PDF]

J Clin Med
Kieć-Wilk B, Guijt P, Dan M, Abdelwahab M, Revel-Vilk S, Serratrice C, International Gaucher Alliance (IGA) and the International Working Group on Gaucher Disease (IWGGD).   +6 more
europepmc   +1 more source

From Lab to Landscape: Environmental Biohybrid Robotics for Ecological Futures

Advanced Robotics Research, EarlyView.
This Perspective explores environmental biohybrid robotics, integrating living tissues, microorganisms, and insects for operation in real‐world ecosystems. It traces the leap from laboratory experiments to forests, wetlands, and urban environments and discusses key challenges, development pathways, and opportunities for ecological monitoring and ...
Miriam Filippi
wiley   +1 more source

New insights in pediatric metabolic medicine: pathogenesis of cholesterol biosynthesis defects and innovative therapies for Pompe disease [PDF]

, 2007
Rossi, Emiliano
core  

Early initiation of enzyme replacement therapy as facilitated by newborn screening improves health outcomes among patients with infantile-onset Pompe disease. [PDF]

Genet Med Open
Desai AK, Rodriguez-Rassi E, Parikh S, Russo RS, Kronn D, Hamm JA, Chang IJ, Ortiz D, McPheron M, Lydigsen H, DeArmey S, Young SP, Kishnani PS.   +12 more
europepmc   +1 more source

Magnetic Micro‐ and Nanorobots for Precision Thrombolysis: Design Strategies and Translational Challenges

Advanced Robotics Research, EarlyView.
Micro‐ and nanorobots for targeted thrombolysis. This perspective elaborates on the clinical indication of blood clot disorders and current limitations for treatment. As a novel, alternative solution, micro‐ and nanorobots can be used to treat and break down thrombi.
Joshua M. Mesfin, Yimo Yan, Nikola Cesarovic, Simone Schuerle   +3 more
wiley   +1 more source

Long-Term Safety and Clinical Outcomes With Olipudase Alfa Enzyme Replacement Therapy in Children and Adolescents With Acid Sphingomyelinase Deficiency. [PDF]

J Inherit Metab Dis
Scarpa M, Diaz GA, Giugliani R, Jones SA, Mengel E, Guffon N, Witters P, Ganesh J, Armstrong NM, Srivastava S, Kim Y.   +10 more
europepmc   +1 more source

Mesenchymal Stromal Cell‐Mediated Intercellular Communication: Mapping the Interactome for Skeletal Muscle Homeostasis and Regeneration

Advanced Science, EarlyView.
Liu et al. define a systems‐level interactome of fibroadipogenic progenitor (FAP)‐mediated signaling in skeletal muscle by integrating single‐cell transcriptomics with FAP depletion‐based perturbation analysis. Functional interrogation using a conditioned media bioassay links predicted signaling to multicellular outcomes, establishing a framework to ...
Xingyu Liu, Edgar E. Perez Carbajal, Yih‐Chii Hwang, Sahil A. Mapkar, Benjamin W. Gilman, Sarah A. Bliss, Lam B. Tran, Kalgi T. Mehta, Jacob O. Banyasz, Ming Yu, Reynold R. Liu, Matthew N. Ly, Christapher S. Morrissey, Michael N. Wosczyna   +13 more
wiley   +1 more source

Successful Pregnancy Outcome in Patient with Pompe Disease Despite Discontinuation of Enzyme Replacement Therapy. [PDF]

Eur J Case Rep Intern Med
Leskovar Lemešić D, Perica D, Zlopaša G, Knezić Frković B, Šućur N, Reiner Ž, Pećin I.   +6 more
europepmc   +1 more source