Results 191 to 200 of about 2,687,044 (278)

Exploratory Study on the Challenges of Newborn Screening for Lysosomal Storage Disorders Emphasizes the Need for Multitier Testing and Collaborative Approaches to Management. [PDF]

JIMD Rep
Terrell A, Sapp K, Graham B, McPheron M, Wetherill L.   +4 more
europepmc   +1 more source

Characterization of Lysosomal Hydrolases and Transporters and Their Age‐Dependent Variability: Relevance to Drug Metabolism and Transport of Small Molecule and Biologic Drugs

Clinical Pharmacology &Therapeutics, EarlyView.
Lysosomes play a key role in the accumulation, catabolism, and transport of endogenous and exogenous metabolites and proteins and are involved in drug metabolism and prodrug activation. However, the protein abundance and interindividual variability of lysosomal drug‐metabolizing enzymes and transporters (DMETs) remain underexplored.
Darshak Gadara, Dilip Kumar Singh, Sandhya Subash, Guihua Yue, Deepak Ahire, Chris Bohl, Maciej Czerwinski, Stephanie Helmstetter, Scott Heyward, Robert S. Jones, Cyrus Khojasteh, Ryota Kikuchi, Priyanka Kulkarni, Bin Ma, Bernard Murray, Chris Seib, Bill Smith, David M. Stresser, Mitchell E. Taub, Ting Wang, Bhagwat Prasad   +20 more
wiley   +1 more source

Lysosomal Storage Disorders

, 2019
Daniel Bell, Sepehr Haghighi, Tristan Skalina   +2 more
openaire   +2 more sources

Mesenchymal Stem Cell-Derived Extracellular Vesicles: Seeking into Cell-Free Therapies for Bone-Affected Lysosomal Storage Disorders. [PDF]

Int J Mol Sci
Leal AF, Pachajoa H, Tomatsu S.
europepmc   +1 more source

From Biological Mechanisms to Causal Inference: Quantitative Systems Pharmacology and Causal Frameworks in Drug Development

Clinical Pharmacology &Therapeutics, EarlyView.
Digital patients and in silico clinical trials offer new opportunities to advance pharmacological research, clinical development, and regulatory decision‐making by enabling the simulation of treatment effects prior to testing in patients. Among the most promising of these opportunities is the combined use of quantitative systems pharmacology (QSP) and ...
Thomas Klabunde, Ramon Hernandez, Piet H. van der Graaf, Tommaso Andreani   +3 more
wiley   +1 more source

Rat models of musculoskeletal lysosomal storage disorders and their role in pre-clinical evaluation of gene therapy approaches. [PDF]

Mamm Genome
Marcó S, Muñoz S, Bosch F, Jimenez V.
europepmc   +1 more source

Membrane lipid metabolism as a regulatory frontier in neural crest biology: Roles for sphingolipids, cholesterol, and lipid rafts

Developmental Dynamics, EarlyView.
Abstract Neural crest cells are multipotent, migratory stem‐like cells essential for vertebrate development that contribute broadly to many tissues including the craniofacial skeleton, peripheral nervous system, and pigment‐producing cells. Their development progresses through phases of induction, specification, delamination, migration, and ...
Allison E. Mancini, Mallorie P. Jenne, Michael L. Piacentino   +2 more
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   +1 more source

Endocannabinoid receptor 2 is a potential biomarker and therapeutic target for the lysosomal storage disorders. [PDF]

J Inherit Metab Dis
Simonaro CM, Yasuda M, Schuchman EH.
europepmc   +1 more source

Selective N‐Functionalization of Pyrazoles

European Journal of Organic Chemistry, EarlyView.
This review traces the evolution of regioselective N‐functionalization strategies for pyrazoles, a key class of nitrogen heterocycles widely used in medicinal chemistry. Due to rapid tautomerism, N‐functionalization often lacks selectivity, leading to poor and unpredictable regioisomeric outcomes.
Lucas Popek, Matthias Schrader, Christopher J. Teskey   +2 more
wiley   +1 more source