Results 71 to 80 of about 12,462 (264)

Infusion rate adjustment in enzyme replacement therapy with pabinafusp alfa for mucopolysaccharidosis II

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aims Enzyme replacement therapy (ERT) for mucopolysaccharidosis II (MPS II) requires long‐term, weekly intravenous infusions often lasting over 3 h each time, which can burden paediatric patients and caregivers and negatively affect their quality of life and treatment compliance.
Kimitoshi Nakamura, Norio Sakai, Hideaki Hirai, Naoko Takasao, Ryo Ibaraki, Tatsuyoshi Yamamoto, Yuji Sato   +6 more
wiley   +1 more source

Discovery of CRBN‐based molecular glue degraders targeting WIZ transcription factor

Bulletin of the Korean Chemical Society, EarlyView.
This work reports the discovery of 9l, a novel cereblon‐based molecular glue degrader which targets the WIZ transcription factor. Compound 9l facilitates WIZ‐CRBN ternary complex formation to degrade WIZ, subsequently inducing γ‐globin expression in HUDEP‐2 cells and highlighting its potential as a sickle cell disease therapy.
Tae‐Jun Kim, Seongu Seong, Jung‐Ae Kim, Taeyeon Kwon, Hyun Jin Kim, Jeong‐Hoon Kim, Jong Yeon Hwang, Jin Hwa Cho, Kyoungmin Choi   +8 more
wiley   +1 more source

Anxiety, Depression, Stress, and Decision-Making Among Orphans and Non-Orphans in Pakistan

Psychology Research and Behavior Management, 2020
Farah Shafiq,1 Sonia Ijaz Haider,2 Shamaila Ijaz1 1Lahore Institute of Special Care and Attention (LISCA-Private Clinic), Lahore, Punjab, Pakistan; 2Department for Educational Development, The Aga Khan University, Karachi, Sindh, PakistanCorrespondence ...
Shafiq F, Haider SI, Ijaz S
doaj  

Nanomaterial‐based immune therapeutic strategies in neurodegenerative diseases

BMEMat, EarlyView.
This review highlights the immunomodulatory potential of nanomaterials (NMs) in treating neurodegenerative diseases (NDs). It focuses on their roles in regulating innate and adaptive immune responses to maintain immune homeostasis. By providing insights into these mechanisms, the review lays the groundwork for innovative NMs therapeutic strategies to ...
Xinru Zhou, Jia Liu, Shulin Lai, Suhan Yin, Bingmin Luo, Longquan Shao, Yiyuan Kang   +6 more
wiley   +1 more source

From Inflammatory Bowel Disease to Cancer: Gut Microbiota–Immune Microenvironment Crosstalk and Natural Product‐Based Therapeutic Opportunities

Cancer Nexus, EarlyView.
ABSTRACT Inflammatory bowel disease (IBD), primarily Crohn's disease and ulcerative colitis, is a chronic relapsing inflammatory disorder of the gastrointestinal tract and an important risk factor for IBD‐associated cancer. Increasing evidence suggests that gut microbiota dysbiosis, epithelial barrier dysfunction, and immune microenvironment remodeling
Xue Zhang, Chongchong Gao, Rui Jiang, Zijian Wu, Jian Bai   +4 more
wiley   +1 more source

Adults with LCH – orphans with an orphan disease [PDF]

Clinical Medicine, 2006
Tatevossian R, Nanduri V, Salotti J, Sargent C, Norton A, Wells A, Pritchard J   +6 more
openaire   +2 more sources

A Systematic Review on Disease‐Modifying Therapies in Parkinsonian Disorders

Clinical Pharmacology &Therapeutics, EarlyView.
Parkinsonian disorders, including Parkinson's disease, Lewy body dementia, multiple system atrophy, and progressive supranuclear palsy, are progressive neurodegenerative conditions with no treatment options to slow disease progression. This systematic review provides an overview of evidence of disease‐modifying therapies that have been evaluated in ...
Pepijn P.N.M. Eijsvogel, Lars M.G. Smits, Philip H.C. Kremer, Geert Jan Groeneveld   +3 more
wiley   +1 more source

اﺿطراب اﻟﺳﻠوك اﻟﺗواﺻﻠﻲ وﻋﻼﻗﺗﮫ ﺑﺎﻹﺳﻧﺎد اﻻﺟﺗﻣﺎﻋﻲ ﻋﻧد اﻟﺗﻼﻣذة اﻷﯾﺗﺎم وﻏﯾرھم )ﺑﺣث ﻣﺳﺗل ﻣن رﺳﺎﻟﺔ ﻣﺎﺟﺳﺗﯾر(

مجلة كلية التربية للبنات, 2019
This study aims at: Finding out the degree of conduct disorder of the orphan and non-orphans pupils in the sample of the study,and finding out the degree of social support of the orphan and non-orphans pupils in the sample of the study,and finding out ...
أ.د.ﺣﺳﯾن ﻧوري اﻟﯾﺎﺳري م.م.رﺟﺎء ﺻدام ﺟﺑر
doaj  

Sensemaking and CSR Character in Multinational Corporations: A Comparative Study of Headquarters and Subsidiary Practices in the UAE

Corporate Social Responsibility and Environmental Management, EarlyView.
ABSTRACT While corporate social responsibility (CSR) scholarship assumes that organizational consistency signals effectiveness, there remains a knowledge gap about how MNCs navigate competing institutional logics between headquarters and subsidiaries. This study investigates how managerial sensemaking mediates the effects of institutional pressures on ...
Charles Antony Diab, Wendy Stubbs
wiley   +1 more source

RORγt‐APCs: The New Masters of Oral Tolerance

Barrier Immunity, EarlyView.
ABSTRACT Oral tolerance is defined by the hypo‐responsiveness of our body to fed antigens, and its failure can lead to immune‐mediated diseases, such as allergy, chronic inflammation and autoimmune diseases. Decades of research have demonstrated that antigen‐presenting cells (APCs) promote oral tolerance by inducing regulatory T cells (Tregs) and/or ...
Thierry Gauthier, WanJun Chen
wiley   +1 more source