Results 21 to 30 of about 288 (101)

Chemical Approaches to Synthetic Drug Delivery Systems for Systemic Applications

Angewandte Chemie International Edition, Volume 61, Issue 49, December 5, 2022., 2022
Over 100 years after Paul Ehrlich's vision of the “magic bullet”, the major challenges in drug delivery remain unchanged: 1) controlling pharmacokinetic and biodistribution drug distribution and clearance in the blood; 2) solubilizing hydrophobic agents, and 3) selectively targeting specific tissues. This Review describes new alternatives for synthetic
Daniel Braatz, Mariam Cherri, Michael Tully, Mathias Dimde, Guoxin Ma, Ehsan Mohammadifar, Felix Reisbeck, Vahid Ahmadi, Michael Schirner, Rainer Haag   +9 more
wiley   +1 more source

Improved attention linked to sustained phenylalanine reduction in adults with early‐treated phenylketonuria

American Journal of Medical Genetics Part A, Volume 188, Issue 3, Page 768-778, March 2022., 2022
Abstract Pegvaliase is approved to reduce phenylalanine (Phe) levels for people with phenylketonuria (PKU). PRISM‐1 (NCT01819727) and PRISM‐2 (NCT01889862) data were analyzed to evaluate the relationship between Phe and inattention in adult participants with PKU.
Deborah A. Bilder, Georgianne L. Arnold, David Dimmock, Mitzie L. Grant, Darren Janzen, Nicola Longo, Mina Nguyen‐Driver, Elaina Jurecki, Markus Merilainen, Gianni Amato, Susan Waisbren   +10 more
wiley   +1 more source

Partial rescue of neuropathology in the murine model of PKU following administration of recombinant phenylalanine ammonia lyase (pegvaliase) [PDF]

, 2017
Pegylated recombinant phenylalanine ammonia lyase (pegvaliase) is an enzyme substitution therapy being evaluated for the treatment of phenylketonuria (PKU).
William L. Zeile, Cooper, Jonathan D., Jonathan D. Cooper, Marc Goldfinger, O'Neill, Charles A., Philip J. Laipis, Zeile, William L., Corado, Carley R., Carley R. Corado, Laurie S. Tsuruda, Laipis, Philip J., Tsuruda, Laurie S., Charles A. O'Neill, Goldfinger, Marc   +13 more
core   +1 more source

Milestones in treatments for inborn errors of metabolism: Reflections on Where chemistry and medicine meet

American Journal of Medical Genetics Part A, Volume 185, Issue 11, Page 3350-3358, November 2021., 2021
Abstract From Sir Archibald Garrod's initial description of the tetrad of albinism, alkaptonuria, cystinuria, and pentosuria to today, the field of medicine dedicated to inborn errors of metabolism has evolved from disease identification and mechanistic discovery to the development of therapies designed to subvert biochemical defects.
Hilary J. Vernon, Irini Manoli
wiley   +1 more source

Cell‐Based Bio‐Hybrid Delivery System for Disease Treatments

Advanced NanoBiomed Research, Volume 1, Issue 10, October 2021., 2021
Bio‐hybrid delivery systems based on integration of living cells and synthetic materials are widely explored for disease and tumor treatment. Herein, the recent studies, prevalent strategies, as well as clinical studies on the topic of cell‐based bio‐hybrid system are introduced.
Chu-Xin Li, Yong-Dan Qi, Jun Feng, Xian-Zheng Zhang   +3 more
wiley   +1 more source

Case-control study about the acceptance of Pegvaliase in Phenylketonuria

Molecular Genetics and Metabolism Reports, 2020
Introduction: Pegvaliase is a novel enzyme substitution therapy approved by the European Drug Administration (EDA) in May 2019 for the treatment of Phenylketonuria (PKU) in adults and children ≥16 years of age.
Johannes Krämer
doaj   +1 more source

THE CONCISE GUIDE TO PHARMACOLOGY 2021/22: Transporters

British Journal of Pharmacology, Volume 178, Issue S1, Page S412-S513, October 2021., 2021
The Concise Guide to PHARMACOLOGY 2021/22 is the fifth in this series of biennial publications. The Concise Guide provides concise overviews, mostly in tabular format, of the key properties of nearly 1900 human drug targets with an emphasis on selective pharmacology (where available), plus links to the open access knowledgebase source of drug targets ...
Stephen P H Alexander, Eamonn Kelly, Alistair Mathie, John A Peters, Emma L Veale, Jane F Armstrong, Elena Faccenda, Simon D Harding, Adam J Pawson, Christopher Southan, Jamie A Davies, Laura Amarosi, Catriona M. H. Anderson, Philip Mark Beart, Stefan Broer, Paul A. Dawson, Bruno Hagenbuch, James R. Hammond, Jules C Hancox, Ken‐ichi Inui, Yoshikatsu Kanai, Stephan Kemp, Gavin Stewart, David T. Thwaites, Tiziano Verri   +24 more
wiley   +1 more source

DISPLAYING PHENYLALANINE AMMONIA LYASES ON BACILLUS SUBTILIS SPORE SURFACE FOR THE TREATMENT OF PHENYLKETONURIA THROUGH ORAL ADMINISTRATION

, 2023
Bacillus subtilis spores are formed in response to adverse conditions as a survival mechanism. These spores are in a dormant state and do not require any nutrients. Their DNA is wrapped with self-assembled multilayers of protective proteins. These spores
Wu, Guo
core  

Inherited metabolic epilepsies–established diseases, new approaches

Epilepsia Open, EarlyView.
Abstract Inherited metabolic epilepsies (IMEs) represent the inherited metabolic disorders (IMDs) in which epilepsy is a prevailing component, often determining other neurodevelopmental outcomes associated with the disorder. The different metabolic pathways affected by individual IMEs are the basis of their rarity and heterogeneity.
Itay Tokatly Latzer, Phillip L. Pearl
wiley   +1 more source

Aging‐Derived Alterations in Genomic, Immune, and Metabolic Networks: Implications for Cancer Development and Therapy

MedComm – Oncology, Volume 5, Issue 1, March 2026.
Ageing acts as a double‐edged sword in cancer. In the elderly, open chromatin, immunosenescence, and chronic inflammation drive SASP (IL‐6, MMPs), MDSC accumulation and T‐cell suppression, fostering tumor‐promoting microenvironments and limited therapeutic benefit.
Qi Wang, Xue Sun, Xinhao Han, Xingda Zhang, Wenzheng Wang, Hongbin Wang, He Ren, Lina Gu   +7 more
wiley   +1 more source