Pharmacological Chaperone definition

Pharmacological Chaperone means a small molecule drug that selectively binds to the active site of a target enzyme resulting in enzyme stabilization, improved trafficking, less aggregation, and/or increased activity of the enzyme.
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Examples of Pharmacological Chaperone in a sentence

  • Co-administration With the Pharmacological Chaperone AT1001 Increases Recombinant Human α-Galactosidase A Tissue Uptake and Improves Substrate Reduction in Fabry Mice.

  • A Pharmacological Chaperone Molecule Induces Cancer Cell Death by Restoring Tertiary DNA Structures in Mutant hTERT Promoters.

  • P.352 A Novel Recombinant Human Acid Alpha-glucosidase, ATB200, Co-administered with a Pharmacological Chaperone, Leads to Greater Substrate Reduction and Improvement in Pompe Disease-relevant Markers Compared to Alglucosidase Alfa in Gaa KO Mice.

  • Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders.

  • First-in-human Preliminary Pharmacokinetic Data on a Novel Recombinant Acid α-Glucosidase, ATB200, Co-administered With the Pharmacological Chaperone AT2221, in Patients With Late-onset Pompe Disease.

  • The Pharmacological Chaperone N- butyldeoxynojirimycin Enhances Enzyme Replacement Therapy in Pompe Disease Fibroblasts.

  • Our Technology Platforms Pharmacological Chaperone TechnologyWe are leveraging our pharmacological chaperone technology to develop next-generation treatments for human genetic diseases by targeting mutated proteins that are unstable, unfolded, or misfolded.

  • Oral Pharmacological Chaperone Migalastat Compared with Enzyme Replacement Therapy in Fabry Disease: 18-Month Results from the Randomized Phase III ATTRACT Study.

  • Disulphide Bridge mediated Repair and Pharmacological Chaperone Rescue RP mutations in the extracellular region of rhodopsin often lead to misfolding and/or instability (Athanasiou et al., 2018).

  • Banning A, Gülec C, Rouvinen J, Gray SJ, Tikkanen R (2016) Identification of Small Molecule Compounds for Pharmacological Chaperone Therapy of Aspartylglucosaminuria.

Related to Pharmacological Chaperone

  • Biological agent shall mean any pathogenic (disease producing) micro-organism(s) and/or biologically produced toxin(s) (including genetically modified organisms and chemically synthesized toxins) which cause illness and/or death in humans, animals or plants.

  • Clinical means having a significant relationship, whether real or potential, direct or indirect, to the actual rendering or outcome of dental care, the practice of dentistry, or the quality of dental care being rendered to a patient;

  • Biological Samples means any physical samples obtained from Study Participants in accordance with the Protocol for the purposes of the Study.

  • Medical cannabis means the same as that term is defined in Section 26-61a-102.

  • Therapeutic diet means meals served that are soft, low-fat, low-sodium or controlled calorie.

  • Wild animal means any mammal, bird, fish, or other creature of a wild nature endowed with sensation and the power of voluntary motion.

  • Biological diversity means the variability among living organisms from all sources including, inter alia, terrestrial, marine and other aquatic ecosystems and the ecological complexes of which they are part; this includes diversity within species, between species and of ecosystems.

  • Cannabinoid edible means food or potable liquid into which a cannabinoid concentrate, cannabinoid extract or dried marijuana leaves or flowers have been incorporated.

  • Diagnostic mammography means a method of screening that

  • Animal means any nonhuman animate being endowed with the power of voluntary action.

  • scientific research means any activity in the field of natural or applied science for the extension of knowledge;

  • Clinical laboratory means a facility for the microbiological, serological, chemical, hematological, radiobioassay, cytological, immunohematological, pathological, or other examination of materials derived from the human body for the purpose of providing information for the diagnosis, prevention, or treatment of a disease or assessment of a medical condition.

  • Opioid antidote means any drug, regardless of dosage amount or method of administration, which has been approved by the United States Food and Drug Administration (FDA) for the treatment of an opioid overdose. “Opioid antidote” includes, but is not limited to, naloxone hydrochloride, in any dosage amount, which is administered through nasal spray or any other FDA-approved means or methods.

  • Bioassay means the determination of kinds, quantities or concentrations and, in some cases, the locations of radioactive material in the human body, whether by direct measurement, in vivo counting, or by analysis and evaluation of materials excreted or removed from the human body. For purposes of these rules, “radiobioassay” is an equivalent term.

  • Precursor means any chemical reactant which takes part at any stage in the production by whatever method of a toxic chemical. This includes any key component of a binary or multicomponent chemical system.

  • Fluoroscopic imaging assembly means a subsystem in which X-ray photons produce a visual image. It includes the image receptor(s) such as the image intensifier and spot-film device, electrical interlocks, if any, and structural material providing linkage between the image receptor and diagnostic source assembly.

  • Study means the investigation to be conducted in accordance with the Protocol.

  • aerodrome means a defined area on land or water (including any buildings, installations and equipment) intended to be used either wholly or in part for the arrival, departure and surface movement of aircraft;

  • Acute toxicity means concurrent and delayed adverse effects that result from an acute exposure and occur within any short observation period, which begins when the exposure begins, may extend beyond the exposure period, and usually does not constitute a substantial portion of the life span of the organism.

  • COVID-19 symptoms means fever of 100.4 degrees Fahrenheit or higher, chills, cough, shortness of breath or difficulty breathing, fatigue, muscle or body aches, headache, new loss of taste or smell, sore throat, congestion or runny nose, nausea or vomiting, or diarrhea, unless a licensed health care professional determines the person’s symptoms were caused by a known condition other than COVID-19.

  • Therapeutic school means a residential group living facility:

  • Assay means a laboratory analysis of Crude Petroleum to include the following: A.P.I. Gravity, Reid vapor pressure, composition, pour point, water and sediment content, sulfur content, viscosity, distillation, hydrogen sulfide, flash/boiling point and other characteristics as may be required by Carrier.

  • Collaborative drug therapy management means participation by an authorized pharmacist and a physician in the management of drug therapy pursuant to a written community practice protocol or a written hospital practice protocol.

  • Formulation means the combination of various ingredients designed to render the product useful and effective for the purpose claimed, or the form of pesticide as purchased by users;

  • Medical cannabis card means the same as that term is defined in Section 26-61a-102.

  • Prosthesis means an artificial substitute for a missing body part.