Natural products: an evolving role in future drug discovery

BB Mishra, VK Tiwari - European journal of medicinal chemistry, 2011 - Elsevier
The therapeutic areas of infectious diseases and oncology have benefited from abundant
scaffold diversity in natural products, able to interact with many specific targets within the cell …

Natural products to drugs: natural product-derived compounds in clinical trials

MS Butler - Natural product reports, 2008 - pubs.rsc.org
Covering: 2005 to 2007 Natural product and natural product-derived compounds that are
being evaluated in clinical trials or are in registration (as at 31st December 2007) have been …

Pompe's disease

AT van der Ploeg, AJJ Reuser - The lancet, 2008 - thelancet.com
Pompe's disease, glycogen-storage disease type II, and acid maltase deficiency are
alternative names for the same metabolic disorder. It is a pan-ethnic autosomal recessive …

Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease

C Spampanato, E Feeney, L Li, M Cardone… - EMBO molecular …, 2013 - embopress.org
A recently proposed therapeutic approach for lysosomal storage disorders (LSDs) relies
upon the ability of transcription factor EB (TFEB) to stimulate autophagy and induce …

Pharmacological chaperone therapy: preclinical development, clinical translation, and prospects for the treatment of lysosomal storage disorders

G Parenti, G Andria, KJ Valenzano - Molecular Therapy, 2015 - cell.com
Lysosomal storage disorders (LSDs) are a group of inborn metabolic diseases caused by
mutations in genes that encode proteins involved in different lysosomal functions, in most …

Iminosugars past, present and future: medicines for tomorrow

G Horne, FX Wilson, J Tinsley, DH Williams… - Drug discovery today, 2011 - Elsevier
Iminosugars comprise the most attractive class of carbohydrate mimetics reported to date
and are ideally positioned to take advantage of our increasing understanding of …

Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics

G Parenti - EMBO molecular medicine, 2009 - embopress.org
Lysosomal storage diseases (LSDs) are a group of genetic disorders due to defects in any
aspect of lysosomal biology. During the past two decades, different approaches have been …

Pharmacological chaperones as therapeutics for lysosomal storage diseases

RE Boyd, G Lee, P Rybczynski… - Journal of medicinal …, 2013 - ACS Publications
Lysosomal enzymes are responsible for the degradation of a wide variety of glycolipids,
oligosaccharides, proteins, and glycoproteins. Inherited mutations in the genes that encode …

Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders

KJ Valenzano, R Khanna, AC Powe Jr… - Assay and drug …, 2011 - liebertpub.com
Many human diseases result from mutations in specific genes. Once translated, the resulting
aberrant proteins may be functionally competent and produced at near-normal levels …

Chaperoning G protein-coupled receptors: from cell biology to therapeutics

YX Tao, PM Conn - Endocrine reviews, 2014 - academic.oup.com
G protein-coupled receptors (GPCRs) are membrane proteins that traverse the plasma
membrane seven times (hence, are also called 7TM receptors). The polytopic structure of …