Results 211 to 220 of about 3,952,882 (246)

Addressing Pancreatic Exocrine Insufficiency and the Impact of Pancreatic Enzyme Replacement Therapy Shortages in Europe. [PDF]

United European Gastroenterol J
Panaitescu-Damian A, Gürcinar I, Stoica V, Paiella S, Hollenbach M, Regel I, Swibold D, Capurso G, Carrato A, Michl P, Arnes L.   +10 more
europepmc   +1 more source

A Rare De Novo Missense Mutation in IFT122 Confers a Genetic Susceptibility Factor of Idiopathic Pediatric Uveitis Via Trio‐based Whole‐Exome Sequencing

Advanced Science, EarlyView.
A rare de novo IFT122‐A773E variant is identified in idiopathic pediatric uveitis and shown to exacerbate retinal inflammation and barrier dysfunction. Mechanistically, the variant enhances IFT43 interaction, elevates calcium signaling, and activates the MEK/ERK/FRA1 axis, revealing a previously unrecognized cilia‐associated pathway that may increase ...
Qian Zhou, Jiaxing Huang, Yilin Wang, Xingran Li, Xianyang Liu, Lingyu Dai, Hongshun Li, Qingfeng Wang, Jiangyi Liu, Guannan Su, Wanyun Zhang, Yang Deng, Qingfeng Cao, Yujie Lai, Xiang Luo, Changwei Huang, Ling Chen, Shengping Hou, Peizeng Yang   +18 more
wiley   +1 more source

Nebulized and intravenous enzyme replacement therapy in mice with mucopolysaccharidosis type II. [PDF]

PLoS One
Shamoun AJ, Reis G, Ashley M, Labilloy A, Ferreira LF.   +4 more
europepmc   +1 more source

Nickel‐Catalyzed Three‐Component Difluoroalkylation‐Amination for the Direct Access to Anti‐Inflammatory β‐Difluoroalkyl Amines

Advanced Science, EarlyView.
A nickel‐catalyzed three‐component reductive fluoroalkylation‐amination strategy of N‐vinylamides for the direct access to the α‐amidyl‐β‐difluoroalkyl amines was reported here. The synthetic method features with broad substrate scope, mild conditions, high functional group tolerance, and convenience to handle.
Chang Xu, Fangting Ma, Fan Ding, Luyan Wang, Yunxi Li, Yunzhi He, Xingang Zhang, Siyu He, Dandan Liu   +8 more
wiley   +1 more source

Long-Term Cardiac Stability Despite Late Enzyme Replacement Therapy in Fabry Disease With Severe Renal Involvement. [PDF]

JACC Case Rep
Teixeira TB, Antunes MO, Paula de Morais Rodrigues Barros A, Brigo IR, Moreira GB, Almeida Torres RV, Gonzales Oliveira Leguizamon JA, Correia VM, Nastari L, Fernandes F.   +9 more
europepmc   +1 more source

Blood Cell‐Camouflaged Liquid Metal Nanoconjugates Orchestrate Treg Depletion and STING‐Amplified Photothermal Immunity for Metastatic Triple‐Negative Breast Cancer Therapy

Advanced Science, EarlyView.
Whole‐blood biomimetic engineering transforms liquid metal nanoparticles into a tripartite immunotherapeutic platform that orchestrates regulatory T‐cell depletion, photothermal‐induced immunogenic cell death, and activation of the stimulator of interferon genes pathway.
Nina Sang, Eijiro Miyako
wiley   +1 more source

Adults With Acid Sphingomyelinase Deficiency Have Sustained Improvements in Clinical Outcomes With up to 5 Years of Olipudase Alfa Enzyme Replacement Therapy: ASCEND Trial Final Results. [PDF]

J Inherit Metab Dis
Wasserstein MP, Hollak CE, Barbato A, Gallagher RC, Giugliani R, Guelbert NB, Hennermann JB, Ikezoe T, Lachmann R, Lidove O, Mabe P, Mengel E, Scarpa M, Senates E, Tchan M, Thurberg BL, Villarrubia J, Armstrong NM, Bhushan S, Kim Y, Kumar M.   +20 more
europepmc   +1 more source

Regulating Cells Fate and Function to Facilitate Bone Regeneration via Designing Programmable Bio‐Interactive Materials

Advanced Science, EarlyView.
This article aims to elucidate the biological mechanisms of bone repair and the evolution of material design, highlighting key cellular and molecular processes. It further proposes strategies and prospects for programmable bio‐interactive materials, which enable precisely guided bone tissue regeneration by dynamically regulating cell behavior and the ...
Qingrui Fan, Fuming Cao, Junqiang Mao, Pengbin Yin, Licheng Zhang, Jianjun Wang, Peifu Tang   +6 more
wiley   +1 more source

Current status of the immunogenicity of enzyme replacement therapy in fabry disease. [PDF]

Orphanet J Rare Dis
Gómez-Cerezo JF, Fernández-Martín J, Barba-Romero MÁ, Sánchez-Martínez R, Hermida-Ameijeiras A, Camprodon-Gómez M, Ortolano S, Lopez-Rodriguez MA.   +7 more
europepmc   +1 more source

Prescribing Patterns and Perceptions of Pancreatic Enzyme Replacement Therapy among Healthcare Providers: A Prospective United States Healthcare Providers Survey. [PDF]

Adv Ther
Ashkar M, Geremia J, Baldwin Z, Edwards ML, Zhang Y, Garcia-Horton V, Yang M, Shen E, Dylla DE, Sanchez Gonzalez Y, Barkin JA.   +10 more
europepmc   +1 more source